Screening for spinal muscular atrophy, the leading genetic cause of infant death, can save lives

2020-11-29T23:01:01

(BPT) – Regina Philipps’ son, Shane, was born a happy, healthy and strong baby — holding his head up on his own, rolling, and pushing himself up on his arms. But at six months old, Regina noticed something might be wrong.

“He started moving less, and some of the strength he had seemed to be disappearing,” said Regina. “It was subtle at first — like when he stopped grabbing my hands and pulling himself up after I changed his diaper. He got to the point where he couldn’t really move his legs. When sitting, he would fall over. He could barely even lift his arms up to his mouth to eat.”

Shane was diagnosed with spinal muscular atrophy (SMA) Type 2 — a progressive, rare genetic disease caused by the lack of a functional survival motor neuron 1 (SMN1) gene.1 Each year, one in every 11,000 babies is born with the disease.2,3 SMA is the leading genetic cause of infant death.1 SMA progresses quickly, meaning the earliest possible diagnosis and treatment is crucial. If left untreated in one of its most severe forms, Type 1, children often require permanent feeding and breathing support or pass away by their second birthday.4 While on average, babies with SMA Type 2 are diagnosed between six and 24 months of age, Shane received his diagnosis at 10 months old and he received treatment soon after.

Millions of babies across the country are routinely screened at birth for conditions that can affect a child’s long-term health or survival. In 2018, SMA was added to the Recommended Uniform Screening Panel (RUSP), a federal list of often devastating disorders that require intervention as early as possible and have treatment options available.5 Despite the fact there are now treatments approved by the U.S. Food and Drug Administration for SMA, only 33 states screen for SMA through permanent or pilot programs, leaving babies in 17 states, plus Washington, D.C., unscreened for this devastating disease.5,6

“We live in New Jersey, which was supposed to implement newborn screening for SMA this August, but because of the COVID-19 pandemic, is implementing it next year. Although we wish we knew sooner, we feel very lucky that Shane’s symptoms were caught relatively early and before he declined even further, so we could pursue treatment as fast as possible,” Regina said. “For babies like Shane, early diagnosis can mean prevention of disease progression. For some babies — especially those with SMA Type 1 — early diagnosis can mean the difference between life and death. That’s why I’m so passionate about educating on the importance of newborn screening, advancing efforts in my state and sharing how people can get involved.”

Children living in states with newborn screening in place can benefit from the remarkable difference early detection and treatment can make — an issue Rory and Carolyn Philstrom know firsthand. It wasn’t long before their son Edan was born that they moved from North Dakota to Minnesota, where Rory was due to start a new job. Because Minnesota actively conducts newborn screening for SMA, Edan’s pediatrician called five days after his birth to tell them his newborn screening test resulted in a positive diagnosis for SMA Type 1. They were blindsided by the diagnosis — but given how quickly SMA progresses, grateful that they could quickly work with their physician on a treatment plan — without losing the precious time they wouldn’t have benefited from in North Dakota. Without screening, children with SMA Type 1 typically receive a diagnosis anywhere up to 6 months after birth.

“Just the sooner you get the diagnosis — the sooner you get that phone call — can make a difference. Even days can make a difference,” said Carolyn.

Since Shane’s diagnosis, Regina has been an active member of the SMA community, helping to advocate for SMA to be added to her state’s newborn screening panel. Their efforts were successful and in January 2020, New Jersey passed a law to screen for SMA, which will be implemented in 2021. Other states have followed, including California, Illinois and Washington. By 2022, Regina, along with patients, caregivers, and the entire SMA community at large, are hopeful every newborn in the United States will be screened for this devastating disease.

For more information about SMA and to inquire about what you can do to ensure that babies in your state are screened for this disease, visit CureSMA.org/ActionCenter.

References

  1. Anderton RS and Mastaglia FL. Expert Rev Neurother. 2015;15(8):895-908.
  2. Sugarman EA, et al. Eur J Hum Genet. 2012;20(1):27–32.
  3. Cure SMA. About SMA. https://www.curesma.org/about-sma/. Accessed June 21, 2020.
  4. Finkel RS, Finkel RS, et al. Neurology. 2014;83(9):810-817.
  5. Cure SMA. Newborn Screening for SMA. https://www.curesma.org/newborn-screening-for-sma/. Accessed November 18, 2020.
  6. Cure SMA. https://www.curesma.org/wpcontent/uploads/2020/02/SMA-State-Fact-Sheet_2020_DC_v3.pdf. Accessed November 18, 2020.

US-ZOL-20-0501

11/2020


Demystifying health coverage and navigating annual enrollment in the health exchange program

2020-11-25T14:09:01

(BPT) – 2020 has been a year like no other. It’s easy to get distracted from our day-to-day lives and responsibilities when there always seems to be a new update related to COVID-19, election results and everything in between. Nevertheless, it is important to be aware of the annual healthcare coverage enrollment period happening now. It is time to secure healthcare coverage and choose a plan that works for you and your family. The deadline to select a plan in most states is Dec. 15 (Jan. 15 in Colorado). You can also enroll outside of this timeframe if you experience certain life events that qualify you for a special enrollment period (SEP).

Last year, approximately 11.4 million people either selected or re-enrolled in a healthcare plan through the exchange program. Furthermore, a report from the Commonwealth Fund estimates that nearly eight million workers have lost jobs with employer-sponsored coverage as of June. Factoring in dependents, this created a healthcare coverage need for 14.6 million Americans in the first four months of the pandemic alone (March-June). With this record level of unemployment, the need for affordable, reliable and accessible healthcare coverage through state-run healthcare exchange plans is more important than ever.

Almost anyone can get covered through a healthcare exchange program. To be eligible you must live in the United States, be a U.S. citizen or national and cannot be incarcerated. However, if you qualify for Medicare or Medicaid, those options may be a better fit for you. And if you have coverage options through your employer, it’s really up to you to decide between that coverage or the plans offered on the exchange.

How to find affordable, reliable and accessible healthcare

With a UnitedHealthcare Exchange plan, you’ll know you will be covered when you need care. UnitedHealthcare plans provide essential coverage along with the affordability of the healthcare exchange.

These plans offer up to three visits and preventative care at no additional cost, as well as low-cost prescriptions. Additionally, they offer access to many healthcare providers, enabling you to easily reach your preferred doctor quickly.

UnitedHealthcare believes that everyone deserves affordable coverage that comes along with expert guidance and support. Call 1-800-806-0451 (TTY 711) or visit www.UHCExchange.com for more information.

UnitedHealthcare has customer service representatives available to help you choose the right plan to fit your needs. From digital care options to personalized support, UnitedHealthcare Exchange plans can help you get the care you need when you need it.

During this time of economic uncertainty amidst a global pandemic, it’s more important than ever to ensure you have healthcare coverage that meets your needs. Hundreds of thousands of Americans have signed up for new plans in the past few months. Make sure you understand your options and select a plan that works for you.

Policies have exclusions, limitations, and terms under which the policy may be continued in force or discontinued. For costs and complete coverage details, contact your insurance agent or UnitedHealthcare.

Medical plan coverage offered by UnitedHealthcare of Arizona, Inc., Optimum Choice, Inc., UnitedHealthcare of Wisconsin, Inc., UnitedHealthcare of Oregon, Inc. and UnitedHealthcare Insurance Co. Administrative Services provided by United HealthCare Services, Inc. or their affiliates.


How to be ready for the next power outage

2020-11-25T12:01:00

(BPT) – Chances are, you’ve been through it — when severe weather hits, there’s nearby construction or just out of the blue, your power suddenly goes out. And you don’t know how long it will last — minutes, hours or days. It’s not just an inconvenience for your family, especially since everyone is spending more time working and schooling from home these days, but it can actually be unsafe. You may need to heat or cool your home so it’s livable, someone in your family may rely on home medical equipment, and you want your refrigerated food to be safe to eat.

Power outages have been increasing due to extreme weather events, plus more people staying home — sometimes causing an overload in energy demands. Being able to cope with unexpected power outages is crucial to your family’s comfort, well-being and safety.

How to be ready for power outages

You may think you’re ready for an outage because you’ve got plenty of flashlights and batteries. Maybe you even have a portable charger for your phone and electronic gadgets. But think about all the items powered by electricity in your home. Even if you have a gas stove, you need electricity to safely ignite it. You rely on your modem to provide internet and even phone connectivity. And the longer the outage lasts, the more you’ve got to worry about.

What your family really needs is safe, reliable backup power.

Choosing a generator for your home

When extreme weather events hit, you don’t want to be one of the many people running to the home improvement store to try to snag the last generator on the shelf. The truth is, you actually have two choices when it comes to generators — a portable model or an automatic standby generator. Either one can help you during an outage, so it’s a good idea to know the difference between the two.

Portable generators

Having a portable generator handy gives you an easy-to-use, low-cost option which offers you some peace of mind in case of a temporary outage. One benefit to portable generators is that they can be used for a variety of outdoor activities like camping and tailgating.

Portable generators supply enough power for a handful of individual products or systems in your home, like your refrigerator or lights. They are manually operated, which means you need to be home to get them started and keep them fueled throughout the power outage.

In addition, to prevent carbon monoxide poisoning, you need to be sure your portable generator is placed at least 20 feet away from your home outdoors, and not inside a garage or screened-in porch. Having a carbon monoxide detector in your home is a highly recommended safeguard.

When using a portable generator, make sure all the systems and appliances you’re backing up from your home are plugged in with properly rated extension cords that run from your home outdoors to the portable generator.

Companies like Kohler offer portable generators in a variety of sizes to meet your power demands. Most portable units run on gasoline, so be sure to keep extra fuel on hand.

Automatic standby generators

For a more permanent solution, you could choose to have an automatic standby generator permanently installed outside your home, similar to a central air conditioning unit. These generators can power everything in your home, and they run on propane or natural gas supplied from your home, with no refueling necessary.

One big advantage of an automatic standby generator is that you do not need to be present to start up and operate the system. When the power goes out, an automatic transfer switch shifts power from the main utility to your generator within seconds — whether you’re home or away.

A Kohler Home Generator delivers high-quality power to your home that won’t harm your sophisticated electronics. Larger units can supply enough power to keep your critical hard-wired systems running, including your HVAC, sump pumps, security systems and large appliances. These generators run quietly and come in a corrosion-resistant housing which prevents rusting. You can also use your smartphone or tablet for remote monitoring of the system.

Want to learn more about protecting your home from the next power outage? Visit KohlerPower.com.


Safety first: Tips for pet-proofing your home

2020-11-25T10:01:00

(BPT) – Welcoming a four-legged friend into your family is an exciting time for everyone, but puppies, kittens, dogs and cats can easily find themselves in serious trouble while exploring their new surroundings. It is important to remember that pets are naturally curious and explore their worlds with their mouths, so they will want to interact with or consume anything left out at their level. Because of this, your home is filled with potential risks, some obvious and others you might not expect. The good news is there are simple steps you can take to help keep your new pet safe.

Purina behavior scientist Dr. Ragen T.S. McGowan offers these timely tips to help pet-proof your home and give you peace of mind that it is a safe place for your dog or cat to live and thrive.

How to Keep Your New Pet Safe

1. Put away the toxins

Many common household items are toxic for pets. Cleaning supplies, detergent, bleach, fertilizer, pest or rodent bait traps and paint can all cause serious harm if ingested. McGowan says, “Make sure to always store toxic items in a place where your pet cannot access them, either in a locked cabinet or on high shelves out of their reach. Even if you are vigilant about keeping these items out of reach, sometimes accidents happen. It is good to have the number for the Animal Poison Control Center handy or even saved in your phone, just in case: (888) 426-4435.”

2. Secure small hardware

Pets are attracted to small items that they can mouth, chew or bat around in play. In the home, small hardware items — from buttons, pins, batteries and magnets, to nails, staples, tacks and paper clips — may look appealing to pets, but could cause serious cuts or even damage internal organs if swallowed. “Before your new friend comes home, do a sweep to ensure small hardware items, batteries and magnets are securely stored or kept out of reach,” shares McGowan. “This means getting down to their level and retrieving any items that may have fallen under or behind the furniture. Tacks, pins and magnets should also be moved up from low hanging bulletin boards or the front of the refrigerator, where they could attract the attention of cats, especially.”

3. Beware of food bags … and practice proper food storage

Almost every one of us is guilty of leaving a bag of chips or box of cereal open on the couch or kitchen table. This may seem harmless, but many food bags, including for pet food, are made from a strong material that helps keep food fresh. A curious pet can get stuck if it goes looking in the bag for leftover crumbs or an early dinner.

“Food packaging can be quite attractive to pets. Not only does it smell like food, but it makes a crinkling noise that pets associate with food, treats or play,” shares McGowan. “Make sure to store food or treat bags up high out of your pet’s reach, or inside cabinets or closets. We also recommend rolling the pet food bag closed and storing the entire item in a resealable hard container, so you can keep the label information handy,” advises McGowan. “A pet of any age, size or strength can quickly find themselves in a troubling, and potentially heartbreaking, situation when left unattended with a food bag. By using resealable containers, you limit the risk — and, as a bonus, your food will remain fresh.” Similarly, remember to cut empty bags along the entire top, bottom and down at least one side before disposal, in case your curious pet rummages through the trash.

4. Conceal electronics

Electronics are ubiquitous in most homes, but the many wires and cords they come with can look like a fun toy to your pet. To keep your pet from chewing them or getting tangled, bundle loose cords behind furniture and store extra chargers and cables in a place that cannot be reached or opened with a paw or nose. For cords that must remain visible, McGowan suggests camouflaging them with cable-coverings or tape, so they are less attractive to your pet. “It is also a good practice to store remote controls and video game controllers in a bin or a drawer where your pet does not have access to them,” she adds. “These items often smell like food or lotions from our fingers which can attract pets to chew on them, but their electrical components and batteries are harmful if consumed.”

5. Keep dangerous ingredients out of reach

In the midst of the COVID-19 pandemic, many people are making more meals at home, including experimenting with new recipes. It’s important to remember that some common ingredients can be harmful to your pets. “Before you make your grocery list, review recipes against a list of ingredients that could be toxic for dogs and cats,” counsels McGowan. “Chocolate is well known, but you’ll also want to avoid grapes, raisins and certain spices. Xylitol, a sugar alternative found in everything from cake mixes to yogurt, peanut butter and chewing gum, is another toxic ingredient.” Best practice is to keep all food and ingredients stored in cupboards, pantries, drawers or on high shelves out of reach of your pet.

We all want to make the most of life with our new furry companions, and by following the above tips, we can do so safely. We also encourage you to have your veterinarian’s contact information easily accessible, so you are ready in case of trouble.

As you prepare to start life with your new pet on the right paw, you can read more tips from McGowan and other Purina experts at www.purina.com/articles.


5 tips for budget-savvy gift shopping

2020-11-25T09:19:00

(BPT) – Are you ready for the holidays? Searching for the perfect gifts for friends and family can be fun and exciting, but it can also be stressful. Especially when money is tight, it’s a challenge to shop for just the right gifts for your loved ones and BFFs. But with a bit of planning, holiday shopping can be just as fun as ever — without clearing out your bank account or piling on credit card debt.

Smart holiday shopping starts with a clear strategy — so get out your tablet or notebook and get started ASAP.

1. Create a budget

Knowing your realistic gift-giving budget can save you headaches down the road. Consider your monthly income and your usual expenses when figuring out how much you can realistically spend on your friends and fam this year.

2. Make a list and check it twice

Your budget got you worried? Consider who you really need and want to shop for — and what they really want.

Maybe your mom would prefer an hour’s worth of Facetime with you so you can chat while you do a craft or your nails together, instead of another knickknack or tchotchke for her dresser. Perhaps your dad would really love a coupon book for doing some yardwork for him, instead of a tie. Think about homemade or handcrafted items your loved ones might really enjoy instead of something you need to buy.

3. Use a smart budgeting app

Want to take advantage of a Cyber Monday deal, but worried about blowing your budget? You can buy now and pay later — with no interest — using a smart budgeting app.

The Quadpay app divvies up the total of your purchase into four equal amounts, one you pay now and the other three installments spread out over the next six weeks, with no interest and no hidden fees — Quadpay even sends helpful reminders of upcoming payments via text message. You can use it to purchase items anywhere, whether it’s online or in-store — with thousands of major retailers to choose from.

The payments will be automatically charged to the account you provide. And if you’re shopping in-store, touch and go with the app for safer, contactless shopping.

The best news? There’s no hard credit check or impact on your credit score. It’s a stress-free way to handle bigger-ticket items you want for your special someone — but need to spread out payments over time to manage your finances smartly.

Download the app from the Apple App Store or Google Play — or visit Quadpay.com to learn more.

4. Keep an eye on the sales

Yes, you can use the Quadpay app to catch the best deals on Black Friday, Small Business Saturday and Cyber Monday. But if you don’t snag the items at the top of your list by then, holiday sales are still likely to show up over the next few weeks. Retailers are pulling out all the stops to entice consumers this year, so keep your eye on prices for items you want both during and after the big name sales days.

Research online for a sense of what regular prices are for these items, so you’ll know a bargain when you see one. Keep a running list, and search online and in-store options where the same or similar products might be available for less.

And don’t panic if a website claims there’s only two left — chances are that’s just a sales tactic to get you to buy now.

5. Shop early

While you may not have seen the perfect deal on that one special item, don’t wait until the last minute to finish your shopping. Most retailers offer free shipping with any purchase, or purchases over a certain amount. If you wait too late, you can run into trouble — you don’t want to have to pay extra for expedited shipping just because the gifts you ordered can’t arrive on time. Avoid shipping fees by giving your loved ones gifts cards if you find the calendar slipping away from you!

Take the time now to plan your strategy. With these tips, you can shop smartly for a much less stressful, much merrier holiday season.


Cool gift ideas that save energy (and do a world of good)

2020-11-25T13:47:00

(BPT) – When checking your holiday list, you’re searching for gifts that inspire joy and delight, and electronic items often fit that description. But what if that gift also happens to have a positive impact on the recipient’s life and the world at large? In these difficult times, many of us have a greater appreciation for the things that protect the health of the planet for everyone.

Whether you’re shopping for a shiny new appliance or gadgets to make someone’s life easier during quarantine, here’s how your gift can do a world of good: Look for products that have earned the ENERGY STAR label. These are independently certified to use less energy than standard products, which means energy bill savings and a cleaner environment.

To inspire you, here are a few holiday shopping list ideas:

Cool savings on the hottest electronic devices: Did you know the average household owns 24 consumer electronics products? The more you invest in items designed to save energy, the more the savings will rack up. That’s especially important now, as families use multiple electronics for work and school. For catching up on favorite shows during times of social distancing, tablets use four times less energy than streaming to a laptop and 15 times less than a desktop computer. An energy-efficient sound bar offers a sleek solution to achieving that rich stereo effect, but uses 70% less energy than a conventional model.

A smarter solution to save heating costs: Now that more people are spending time at home, they’re spending less on dining and gasoline. However, home energy costs have definitely skyrocketed. Smart thermostats take the guesswork out of maximizing energy efficiency, and choosing an ENERGY STAR certified smart thermostat means savings you can trust. Were everyone to make the switch, we’d save 56 trillion BTUs of energy and $740 million a year, offsetting 13 billion pounds of annual greenhouse gas emissions.

Appliances with noticeable cost savings: Even when the purchase price is nice, don’t overlook this hidden cost of owning appliances: The cost of operating the product. So, if you’re eyeing a new washer dryer set as the perfect holiday gift, make sure it’s energy efficient. You’ll feel good helping your loved one realize more than 20% savings on their electric bill while using one-third less water compared to the standard models.

A bright idea for stocking stuffers: Here’s a small gift that can light up their lives and save some serious bucks. A single energy-efficient light bulb can save more than $50 in electricity costs over its lifetime. If each American home replaced just a single bulb to one that earned the ENERGY STAR certification, we’d prevent more than 7 billion pounds of greenhouse gas emissions.

Now that you know how ENERGY STAR-certified appliances and devices can do a world of good, for the recipient as well as our planet, be sure and check out the handy gift guide by visiting energystar.gov/holiday.


Seasonal Holiday Martini Recipes [Infographic]

2020-11-25T10:15:01

While the holidays look a little different this year, one thing remains consistent: a seasonal cocktail elevates the occasion. The martini is the perfect cocktail for the holiday season, and The Botanist Islay Dry Gin makes the perfect martini.

(BPT) –


4 ways to protect your finances and investments in 2021

2020-11-25T08:01:01

(BPT) – Finances are consistently a top concern for many Americans, with “saving money” a top-10 most common New Year’s resolution. This year, Americans are more concerned than ever before due to the uncertainty created by the COVID-19 pandemic. USE Credit Union reported that more than 75% of non-transactional calls received since the start of the pandemic were from members concerned about their financial future, citing economic hardship as the primary reason for concern. The economy and job market remain in a state of constant flux, which is causing many families to worry about their ability to pay an unexpected bill, continue to pay off student loans, mortgages or credit card debt, or save money for the future.

“Saving money is more than just putting spare change into a coffee can, or simply ordering takeout less often,” said Jeff Schroeder, vice president and chief product officer at Mercury Insurance. “Sure, those things can add up over time, but people may find that their greatest savings can come from taking a look at the necessary expenses they pay for every month, such as insurance.”

Schroeder recommends these four tips to help protect your finances in the coming year:

1) Check your auto insurance coverages. There’s no reason to pay for more coverage than you need, but being underinsured can leave you exposed. “The cost of repairs after a collision has grown in recent years, both as a result of more crossovers and SUVs on the road, and more technologically advanced vehicles,” said Schroeder. “Beyond paying for more expensive repairs if your insurance doesn’t cover it, if you’re underinsured, you may also be responsible for paying out of pocket for medical bills, which could potentially devastate savings for a down payment on a house, your child’s college tuition or a future vacation. It’s vitally important to make sure you have the right amount of auto insurance coverage to protect against unforeseen events.”

2) Know what your homeowners insurance covers. First and foremost, be sure to read your homeowners insurance policy so you’re clear about what it does and doesn’t cover. It’s a good idea to check in with your insurance agent each year to ensure you have adequate coverage, especially if you’ve made renovations, own collectible or valuable items, or live in an area that’s prone to flooding or earthquakes, as standard homeowners insurance policies typically don’t cover these situations. Also, maintain a home inventory to make sure to have an accurate record of your belongings and property.

3) Be aware of potential gaps in coverage. A standard homeowners insurance policy often doesn’t cover mechanical failures to your home’s appliances, HVAC or other essential systems, nor does it cover a break to service lines on your property that supply your home with electricity, gas or sewer functions. In either of these scenarios, this means you would be responsible for writing a big check to a repair company or having to purchase a pricy replacement. However, adding home systems protection and service line protection endorsements can help provide coverage for costly repairs and replacements, saving money and your peace of mind. Pennies spent now can save you thousands of dollars later.

4) Regularly shop for the best coverage and price. Insurance prices can vary significantly from company to company, so it’s a good idea to take a few minutes to see if you’re getting a good deal. Shop around at least once a year — making sure to look for the exact same coverage limits — to see if you can find a more affordable rate.

“Often, regional insurers like Mercury Insurance are more attuned to their policyholders’ needs and can offer better rates,” Schroeder added. “For example, the California Department of Insurance found that Mercury auto insurance policyholders save an average of $670, which can go a long way in feeling more secure in your savings.”

The most effective way to make sure your finances are minimally impacted by insurance costs in 2021 is to speak to an independent insurance agent. They can help make sure you have the proper amount and type of coverage to keep yourself, your family and property protected.


Achieving sense of stability following diagnosis – one woman’s journey with a rare neurological disease

2020-11-24T23:01:00

(BPT) – Sally is a second grade teacher who loves spending time with her family and being outdoors — she has always been healthy and active, enjoying hikes and practicing yoga. Four years ago, she started experiencing a strange pain in her right arm and hand.

“It felt like a combination of numbness and shooting pain,” describes Sally. “I thought I had a pinched nerve, so I made an appointment with my physician to figure out what was wrong.”

Difficulties with diagnosis

Her first doctor’s visit resulted in an unexpected diagnosis — Sally was told that she had suffered a stroke, which did not make sense to her. Questioning the diagnosis and wanting more clarity about her health, she met with a neurologist, requesting an MRI to evaluate her cervical spine. The MRI showed lesions on her spine, which led to another diagnosis: multiple sclerosis (MS). As her arm numbness worsened, Sally’s care team worked to determine her specific type of MS and discuss potential treatment options.

First treated with oral prednisone, Sally’s symptoms did not improve, and she experienced non-stop vomiting for 24 hours. She later learned this can be a symptom of a rare disease — neuromyelitis optica spectrum disorder (NMOSD), but at the time, she was still being treated for MS. When the oral prednisone did not alleviate any symptoms, her care team tried IV steroids next, which helped improve the numbness, but the pain continued. Sally’s neurologist pursued further testing — a spinal tap and blood test — to understand why no treatments were successful. These tests showed the presence of aquaporin-4 antibodies, which are associated with NMOSD but not present in patients with MS. About three months after her initial symptoms, she was accurately diagnosed with NMOSD.

“It was scary receiving a diagnosis for a disease I had never heard of,” recalls Sally. “Learning more about the condition and the progressive nature of the disease left me feeling uncertain and worried about how this diagnosis would impact my future.”

NMOSD can place a heavy burden on patients and their families as attacks and disease severity are unpredictable and could lead to permanent disability.

What is NMOSD?

Neuromyelitis Optica Spectrum Disorder (NMOSD) is a rare, debilitating disease of the central nervous system characterized by immune-mediated damage to the optic nerve, brain stem and spinal cord. NMOSD may cause a wide range of symptoms including permanent blindness, muscle weakness, paralysis, pain and fatigue. The variety of ways that NMOSD manifests may cause delays in diagnosing the disease.

NMOSD affects approximately 10,000-15,000 people in the U.S., and the median age of onset is 40 years (onset ranges from ages 3 to 80).[1],[2] The incidence rate may be up to 9 times as high for women as for men, and the prevalence of the disease is around 2-3 times higher in Black and Asian populations.[3],[4]

The journey to wellness

“The numbness and shooting pain I was dealing with was due to inflammation of the spinal cord,” said Sally. “My neurologist explained to me that NMOSD can also cause inflammation of the optic nerve, causing vision problems and eye pain, including sudden blindness. Fortunately, I have never experienced any symptoms in my eyes.”

After her diagnosis, Sally was referred to an NMOSD Center of Excellence and learned about the different treatments available and although there were no FDA-approved treatments at the time, Sally was enrolled in the N-MOmentum clinical trial for Uplizna® (inebilizumab-cdon). Uplizna, which was approved by the FDA in June 2020, is the first and only B-cell depleter approved for the treatment of NMOSD in adult patients who are AQP4 antibody positive.

“My physician explained that Uplizna is designed for patients with NMOSD and works by depleting the specific B cells that are believed to cause the disease,” Sally said. “After learning more about the drug and talking with my physician, it felt like the right course of treatment for me and that it would fit into my lifestyle.”

Since beginning her treatment with Uplizna, Sally hasn’t experienced any side effects, and most importantly, she hasn’t had any additional NMOSD relapses. “While I still take certain precautions knowing that I have a suppressed immune system, I have been able to continue doing the things I enjoy,” says Sally.

This story is Sally’s personal experience with Uplizna. The most common side effects of Uplizna include urinary tract infections, joint pain, headaches and back pain, and Uplizna may cause serious side effects, including low blood-cell count. Always discuss with your doctor the right treatment option for you.

How NMOSD differs from MS

As in Sally’s case, NMOSD may be difficult to diagnose, because its wide range of symptoms overlap with other illnesses, especially MS. In fact, 41% of NMOSD patients reported receiving an initial misdiagnosis of MS.[5],[6]

Differences between NMOSD and MS:

NMSOD

  • Severe acute episodes can lead to permanent disability
  • Permanent central nervous system (CNS) damage
  • 70% of people with NMOSD are aquaporin-4 antibody seropositive

MS

  • Cumulative effect of individual episodes typically cause disability
  • Often experience better recovery from attacks
  • AQP4 antibody seronegative

NMOSD and MS are treated in different ways, and early detection and treatment help ensure the best outcomes. Differential diagnosis of NMOSD and MS is important, because MS treatments may be ineffective or may exacerbate NMOSD.[7],[8]

Important Safety Information

What is Uplizna?

Uplizna is a prescription medicine used to treat adults with neuromyelitis optica spectrum disorder (NMOSD) who are anti-aquaporin-4 (AQP4) antibody positive.

It is not known if Uplizna is safe or effective in children.

Who should not receive Uplizna?

You should not receive Uplizna if you have:

  • had a life-threatening infusion reaction to Uplizna.
  • an active hepatitis B virus infection.
  • active or untreated inactive (latent) tuberculosis.

Before receiving Uplizna, tell your healthcare provider about all of your medical conditions, including if you:

  • have or think you have an infection.
  • have ever taken, currently take, or plan to take medicines that affect your immune system, or other treatments for NMOSD. These medicines may increase your risk of getting an infection.
  • have or have ever had hepatitis B or are a carrier of the hepatitis B virus.
  • have or have ever had tuberculosis.
  • have had a recent vaccination or are scheduled to receive any vaccinations. You should receive any required vaccines at least 4 weeks before you start treatment with Uplizna.
  • are pregnant or plan to become pregnant. It is not known if Uplizna will harm your unborn baby. Females should use birth control (contraception) during treatment with Uplizna and for 6 months after your last infusion of Uplizna.
  • are breastfeeding or plan to breastfeed. It is not known if Uplizna passes into your breast milk. Talk to your healthcare provider about the best way to feed your baby if you receive Uplizna.

Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.

What is the most important information I should know about Uplizna?

Uplizna may cause serious side effects, including:

Infusion reactions. Uplizna can cause infusion reactions that can be serious or may cause you to be hospitalized. You will be monitored during your infusion and for at least 1 hour after each infusion of Uplizna for signs and symptoms of an infusion reaction. Tell your healthcare provider right away if you get any of these symptoms:

  • headache
  • sleepiness
  • fever
  • rash
  • nausea
  • shortness of breath
  • muscle aches

If you develop an infusion reaction, your healthcare provider may need to stop or slow down the rate of your infusion and treat your symptoms.

Infections. Infections can happen during treatment with Uplizna. Tell your healthcare provider right away if you have an infection or get any of these symptoms:

  • painful and frequent urination
  • nasal congestion, runny nose, sore throat, fever, chills, cough, body aches
  • Uplizna taken before or after other medicines that weaken the immune system may increase your risk of getting infections.
  • Hepatitis B virus (HBV) reactivation. Before starting treatment with Uplizna, your healthcare provider will do blood tests to check for hepatitis B viral infection. If you have ever had hepatitis B virus infection, the hepatitis B virus may become active again during or after treatment with Uplizna. Hepatitis B virus becoming active again (called reactivation) may cause serious liver problems, including liver failure or death. Your healthcare provider will monitor you if you are at risk for hepatitis B virus reactivation during treatment and after you stop receiving Uplizna.
  • Progressive Multifocal Leukoencephalopathy (PML). PML may happen with Uplizna. PML is a rare brain infection that leads to death or severe disability. Symptoms of PML may get worse over days to weeks. Call your healthcare provider right away if you get any of these symptoms:
    -weakness on one side of the body
    -changes in your vision
    -confusion
    -loss of coordination in your arms and legs
    -changes in thinking or memory
    -changes in your personality
  • Tuberculosis (TB). TB is caused by an infection in the lungs. Before starting treatment with Uplizna, your healthcare provider will check to see if you are at risk for getting TB or have ever had TB.
  • Vaccinations. Certain vaccines, called “live” or “live attenuated” vaccines, are not recommended in people receiving Uplizna. Talk to your healthcare provider before receiving any vaccinations. If you have a baby and you were receiving Uplizna during pregnancy, it is important to tell your baby’s healthcare provider about your Uplizna use so they can decide when your baby should receive any vaccine.

See “What are the possible side effects of Uplizna?” for more information about side effects.

How will I receive Uplizna?

  • Uplizna is given through a needle placed in a vein (IV or intravenous infusion) in your arm.
  • Before treatment with Uplizna, your healthcare provider will give you a corticosteroid medicine, an antihistamine, and a fever prevention medicine to help infusion reactions become less frequent and less severe. See “What is the most important information I should know about Uplizna?”
  • Your first dose of Uplizna will be given as 2 separate infusions, 2 weeks apart.
  • Your next doses of Uplizna will be given as one infusion every 6 months.
  • Each infusion will last about 1 hour and 30 minutes. After each infusion, you will be monitored by a healthcare provider for at least 1 hour.

What are the possible side effects of Uplizna?

Uplizna may cause serious side effects, including:

  • See “What is the most important information I should know about Uplizna?”
  • low blood cell counts. Uplizna may cause a decrease in some types of blood cells. Your healthcare provider will do blood tests to check your blood cell counts.

The most common side effects include urinary tract infection and joint pain. These are not all the possible side effects of Uplizna.

Call your doctor for medical advice about side effects. You may report side effects to the FDA at 1-800-FDA-1088.

For more information and the full Prescribing Information, visit www.Uplizna.com.

[1] Hamid SHM, et al. What proportion of AQP4-IgG-negative NMO spectrum disorder patients are MOG-IgG positive? A cross sectional study of 132 patients. J Neurol. 2017;64(10):2088–2094.

[2] Mealy MA, Wingerchuk DM, Greenberg BM, Levy M. Epidemiology of Neuromyelitis Optica in the United States. Arch Neurol. 2012;69(9):1176-1180.

[3] Wingerchuk DM. J Neurol Sci. 2009;286(1-2):18-23).

[4] Flanagan EP, et al. Epidemiology of aquaporin-4 autoimmunity and neuromyelitis optica spectrum. Ann Neurol. 2016;79(5):775-783.

[5] Beekman, J, et al. Neuromyelitis optica spectrum disorder: patient experience and quality of life. Neurol Neuroimmunol Neuroinflamm, 2019;6(4).

[6] Neuromyelitis optica spectrum disorder. Retrieved from: https://wearesrna.org/living-with-myelitis/disease-information/neuromyelitis-optica-spectrum-disorder/.

[7] Eaneff S, et al. Patient perspectives on neuromyelitis optica spectrum disorders: Data from the Patients Like Me online community. Mult Scler Relat Disord, 2017;17:116-122.

[8] Wingerchuk DM, et al. International consensus diagnostic criteria for neuromyelitis optica spectrum disorders. Neurology, 2015;85(2):177-1.


Cancer Doesn’t Stop: Cycle for Survival Advances Rare Cancer Research With Over $40 Million Raised This Year

2020-11-24T15:01:01

(BPT) – It was in the fall of 2007 when Nicole, 26, felt a large mass in her abdomen. At first she ignored it, hoping it was nothing; she was a healthy, active woman in her 20s. However, when the mass didn’t go away, she realized she needed to see a doctor. In January 2008, after many tests and scans, Nicole was hit with life-altering news: She was diagnosed with a rare incurable cancer called GIST, a type of sarcoma. Thirteen years later Nicole is still here to share her story.

After a few years seeking treatment for her disease at various institutions, Nicole ultimately decided to receive treatment at Memorial Sloan Kettering Cancer Center (MSK) in New York City. This was the beginning of multiple doctor visits and surgeries, including one to remove more than a dozen tumors and a third of her liver. In 2017, almost 10 years since her diagnosis, Nicole participated in a clinical trial with the goal of slowing or maybe even stopping the progression of her disease. This particular trial was almost entirely funded by Cycle for Survival, a national movement owned and operated by MSK that raises critical funds for rare cancer research.

About 50 percent of patients living with cancer have a rare form of the disease. Even though these diseases account for about half of all cancer diagnoses when combined, research on rare cancers is drastically underfunded, often leaving patients like Nicole with limited or no treatment options. This can be devastating to so many, but with the help of Cycle for Survival there is hope. Since 2007, Cycle for Survival has raised over $260 million, with 100 percent of every dollar going to research led by MSK. In 2020 alone, because of the extraordinary Cycle for Survival community and its founding partner, Equinox, the movement raised over $40 million to support a variety of initiatives across many rare cancer types. Doctors who receive these funds credit Cycle for Survival with launching and advancing trailblazing projects that otherwise wouldn’t have had the resources needed to get off the ground. In 2020, Cycle for Survival funded research for over 40 rare cancers, including:

  • Pediatric cancers
  • Brain cancer
  • Blood cancers
  • Gynecologic cancers
  • Sarcoma
  • Bladder cancer
  • Gastric neuroendocrine tumors
  • Kidney cancer
  • Mesothelioma
  • Pancreatic cancer
  • Liver cancer
  • Gallbladder cancer

Nicole’s oncologist and nine-year Cycle for Survival participant Dr. William Tap, Chief of the Sarcoma Medical Oncology Service at MSK, is one of the doctors who received these crucial funds. “Cycle for Survival is our lifeline, our community, our strength. It has become a movement that is changing care paradigms for people with rare cancers,” he says.

Today, Nicole’s tumors are still growing, and there is no treatment or cure for GIST. However, clinical trials similar to the one she was on in 2017 keep her optimistic because she knows Cycle for Survival will continue to fund new and different treatment options for her and other patients worldwide, allowing them to live longer, fuller lives. “Every dollar that we raise truly makes me feel stronger because I have more hope that someday there will be a cure that will keep me going,” Nicole says.

The Cycle for Survival community will never stop fighting until all people with cancer have effective treatment options. Registration for the 2021 Cycle for Survival season opens in December, and the movement will be celebrating its 15th year. There will be many exciting new ways people can get involved, including Cycle for Survival’s first-ever virtual event experience. To learn more, visit www.cycleforsurvival.org.

MORE INFORMATION

About Cycle for Survival

JOIN THE BATTLE. Cycle for Survival is the movement to beat rare cancers. Since 2007, Cycle for Survival has raised over $260 million to fund pioneering rare cancer research led by Memorial Sloan Kettering Cancer Center, which owns and operates Cycle for Survival. 100 percent of every donation funds rare cancer research. About half of all people with cancer are fighting a rare cancer, and they often face limited or no treatment options. Together with the movement’s founding partner, Equinox, Cycle for Survival has supported innovative clinical trials, research studies and major scientific initiatives. For research updates and additional information, visit https://www.cycleforsurvival.org and follow Cycle for Survival on Facebook, Twitter, Instagram and YouTube.

About Memorial Sloan Kettering (MSK)

As the world’s oldest and largest private cancer center, Memorial Sloan Kettering has devoted more than 135 years to exceptional patient care, influential educational programs and innovative research to discover more effective strategies to prevent, control and, ultimately, cure cancer. MSK is home to more than 20,000 physicians, scientists, nurses and staff united by a relentless dedication to conquering cancer. Today, we are one of 51 National Cancer Institute-designated Comprehensive Cancer Centers, with state-of-the-art science and technology supporting groundbreaking clinical studies, personalized treatment, and compassionate care for our patients. We also train the next generation of clinical and scientific leaders in oncology through our continually evolving educational programs, here and around the world. Year after year, we are ranked among the top two cancer hospitals in the country, consistently recognized for our expertise in adult and pediatric oncology specialties. www.mskcc.org.

About Equinox

IT’S NOT FITNESS. IT’S LIFE: Founded in 1991, Equinox was built on the notion that fitness can empower a life well-lived and foster a strong community of high-performance individuals. Nearly 30 years later, Equinox continues to lead and disrupt the category it singularly defined, operating over 100 full-service Clubs globally across major US cities including New York, LA, Miami and San Francisco as well as London, Toronto and Vancouver, each featuring bespoke design in an unparalleled luxury environment. Grounded in the core tenets of Movement, Nutrition and Regeneration and backed by a Health Advisory Board of industry-leading experts across these disciplines, the company offers an integrated membership model Equinox+, which fuses the digital and physical Equinox experiences. The dynamic membership builds upon the touted in-club offering with the launch of virtual personal training and yoga, nationwide outdoor fitness clubs Equinox+ In The Wild, The Daily Check-In, and Variis, a first-of-its-kind multi-brand digital platform offering premium fitness content across its portfolio of brands. In July 2019, Equinox unveiled Equinox Hotels as a true culmination of its lifestyle brand promise, redefining the luxury hospitality experience to be a seamless extension of high-performance living. For more information, visit www.equinox.com.