Author: admin

2024-12-04T08:01:00

(BPT) – Every year, nearly half a million people in the U.S. suffer from a serious and potentially deadly infection called Clostridioides difficile or C. diff.^1,2 C. diff is a bacteria that can overwhelm good bacteria in the gut and cause an infection. Symptoms may include constant diarrhea, stomach pains, and fever.¹ Even when treated with antibiotics, a C. diff infection can come back again and again.¹

Imagine having C. diff and needing to be close to a bathroom almost constantly – it can make even the simplest task like going to the grocery store difficult.¹ That’s what happened to Heather, a 37-year-old newlywed from Arizona.

Heather recalls her fear when she began going through a vicious cycle of symptoms right before her wedding. “I had dreamed of being this beautiful princess bride, and suddenly I was sick all the time. It was devastating.”

Heather was initially treated with antibiotics, but her infection returned. This is common for some C. diff sufferers. Up to three in 10 people who have C. diff get it again, which is known as a recurrence.^2,3 After a first recurrence, up to six in 10 people may develop a subsequent recurrence. ^4,5

When Heather visited her doctor for a follow-up, she learned about an option called REBYOTA, the first and only single-dose microbiome-based therapy approved by the U.S. Food and Drug Administration (FDA) to prevent recurrent C. diff infection in individuals 18 years of age and older following antibiotic treatment for recurrent C. diff infection. REBYOTA is delivered directly where it’s needed, the gut microbiome.

“I went in, and my nurse administered REBYOTA. It only took about 15 minutes, and then I was able to get up and drive myself home,” she said.

A few days after the administration, Heather started to notice a difference.

“I was so surprised. The number of times I was rushing to the bathroom decreased, and each day I felt incrementally better,” she said. “After being sick for so long, I was finally starting to feel like myself again.”

More than six months later, Heather remains free of C. diff symptoms. She even felt well enough to go to the Phoenix State Fair with her husband. “We were there from morning until night, and I didn’t have to worry about being close to a bathroom. It was something I never thought would be possible when I was sick,” she said. Individual results may vary.

To learn more about C. diff infection and REBYOTA, talk to your doctor and visit REBYOTA.com.

INDICATION

REBYOTA (fecal microbiota, live – jslm) is indicated for the prevention of recurrence of Clostridioides difficile (C. diff) infection in individuals 18 years of age and older, following antibiotic treatment for recurrent C. diff infection.

Limitation of Use
REBYOTA is not indicated for the treatment of C. diff infection.

Important Safety Information

• You should not receive REBYOTA if you have a history of a severe allergic reaction (e.g., anaphylaxis) to REBYOTA or any of its components.
• You should report to your doctor any infection you think you may have acquired after administration.
• REBYOTA may contain food allergens.
• Most common side effects may include stomach pain (8.9%), diarrhea (7.2%), bloating (3.9%), gas (3.3%), and nausea (3.3%).
• REBYOTA has not been studied in patients below 18 years of age.
• Clinical studies did not determine if adults 65 years of age and older responded differently than younger adults.

You are encouraged to report negative side effects of prescription drugs to FDA. Visit www.FDA.gov/medwatch or call 1-800-332-1088.

Please click to see the full Prescribing Information.

References:

1. Centers for Disease Control and Prevention. About C. Diff? 6 Mar. 2024. Available at: https://www.cdc.gov/c-diff/about/index.html.
2. Lessa FC, Mu Y, Bamberg WM, et al. Burden of Clostridium difficile infection in the United States. N Engl J Med. 2015;372(9):825-834.
3. Cornely OA, Miller MA, Louie TJ, Crook DW, Gorbach SL. Treatment of first recurrence of Clostridium difficile infection: fidaxomicin versus vancomycin. Clin Infect Dis. 2012;55(suppl 2):S154-S161.
4. Kelly CP. Can we identify patients at high risk of recurrent Clostridium difficile infection? Clin Microbiol Infect. 2012;18(suppl 6):21-27.
5. Smits WK, Lyras D, Lacy DB, Wilcox MH, Kuijper EJ. Clostridium difficile infection. Nat Rev Dis Primers. 2016;2:16020.

Photo by Julian Dufort

2024-12-04T08:01:00

(BPT) – For more than a century, Johnson & Johnson has been synonymous with innovation in science and medicine by moving the needle in building a world where complex diseases are prevented and cured, treatments are smarter, and solutions are personalized and accessible. Inventions like robotic surgery, tissue engineering, and treatments for cancers and HIV have changed the world forever and improved the patient experience and health and care for all.

As an authority on transformative scientific research and discoveries, Johnson & Johnson recognizes the impact scientific research and discoveries can have on the world and is proud to celebrate those innovations — both inside the company and from other scientists around the world — through two awards: the Johnson Medal and the Dr. Paul Janssen Award.

Established in 1960, The Johnson Medal is Johnson & Johnson’s most prestigious company-wide honor and celebrates trailblazing innovation in research and development. The Medal is awarded to teams who exemplify excellence through their curiosity, impactful contributions to patients, collaborative spirit, and unwavering commitment to pushing boundaries and making breakthroughs that accelerate science through novel healthcare solutions.

Johnson & Johnson employees who have previously received this honor include:

• Jill Giles-Komar, David Shealy, Jacqueline Benson, Yevgeniya Orlovsky, Jeffrey Luo and Carrie Brodmerkel, the team that developed a treatment for plaque psoriasis which has since been approved for irritable bowel diseases and psoriatic arthritis
• Dr. Assaf Govari, a four-time winner who created several medical technological devices, including an ultrasound that provides real-time, 3D imaging of the inside of the heart
• Jordan Schecter, Arnob Banerjee, Jean Xu, Jennifer Yohrling, Loreta Marquez and Ravi Bhatia for developing J&J’s first CAR-T cell therapy for treating multiple myeloma
• Thorsten Siess, Claudia Mourran, Frank Kirchhoff, Jimpo Wang, Scott Corbett, Vincent Roy and Werner Carell who developed the only heart pump to help restore native heart function during surgery or following a heart attack

Passionate and creative scientists are all around us and in an effort to celebrate the impact these gifted researchers can have on transforming human health and inspiring others, Johnson & Johnson also honors a scientist or research team whose achievements have made a measurable impact on health for all with the Dr. Paul Janssen Award for Biomedical Research.

Established in 2004, this award honors the memory of Dr. Paul Janssen, a passionate, prolific scientist and researcher who discovered over 80 treatments — four of which remain on the World Health Organization’s list of essential medicines — for many illnesses, including infectious diseases, pain, and psychiatric disorder. The Award extends the legacy of its namesake and lauds groundbreaking biomedical researchers outside of Johnson & Johnson who have made “a transformational contribution toward the improvement of human health.” More than a third of this award’s winners have received a Nobel Prize for their foundational work.

Counted among the award’s recipients are:

• Dr. Emmanuelle Charpentier and Dr. Jennifer Doudna for their co-discovery of a new method for precise genetic information manipulation
• Dr. Erik De Clercq and Dr. Anthony Fauci for their work in understanding and combatting viral diseases, particularly HIV/AIDS
• Dr. Robert Langer for his development of novel drug delivery systems that have revolutionized treatment for a number of diseases and chronic conditions
• Dr. Lynne Maquat and Dr. Alexander Varshavsky for their fundamental discoveries about regulated degradation of RNA and proteins

Johnson & Johnson is committed to honoring scientific creativity and excellence that can improve the lives of patients around the world. These awards aim to inspire future generations of scientists and researchers to continue to push boundaries through cutting-edge science and transformative healthcare solutions while honoring the legacies of the pioneers who came before. Johnson & Johnson knows that recognizing and supporting innovation — wherever it originates — is key to advancing medical discoveries. As Dr. Paul would say, “There is so much more to be done; the patients are waiting.”

2024-12-04T10:55:00

(BPT) – Tatiana loves hiking, training their service dog, and attending open-mic nights and poetry slams. When Tatiana was unable to stay awake and started falling asleep in college classes, they knew there was something wrong.

The excessive daytime sleepiness that Tatiana was experiencing was diagnosed as narcolepsy. Narcolepsy is a rare neurological disorder that can affect all aspects of a person’s daily life, including fighting to stay awake throughout the day, falling asleep without warning, or feeling sleepy in situations that are less active such as watching TV or riding in a car. It is estimated that 170,000 people in the United States live with narcolepsy.

Asim Roy, MD, who is the Medical Director of the Ohio Sleep Medicine Institute and a board-certified sleep specialist and neurologist, said, “Narcolepsy is often undiagnosed or misdiagnosed. Many people experience symptoms of narcolepsy for years before receiving an accurate diagnosis of narcolepsy.”

Living with narcolepsy and common misconceptions

All people living with narcolepsy experience excessive daytime sleepiness, which is defined as the inability to stay awake and alert during the day or the persistent feeling of tiredness. For Tatiana, excessive daytime sleepiness was being unable to stay awake in sedentary situations like sitting down in class or waiting for appointments, which became a very big obstacle.

“Unfortunately, there are several misconceptions about people living with narcolepsy, many of which are not true. Narcolepsy symptoms can be hard for others to fully understand; therefore, people living with narcolepsy can experience stigma as a result of this,” Dr. Roy explained. “Although we all get tired, people living with narcolepsy feel an overwhelming sense of sleepiness and have to fight to stay awake and alert throughout the day, and may even fall asleep without warning,” he added. “This can be hard for people because of the impact it can have not only on their own daily life, but also their relationships with others.” Dr. Roy added, “Their excessive daytime sleepiness can cause them to withdraw socially and arrive late or fully miss events, which can further lead to strained relationships if not understood by others.”

Searching for treatment options

Tatiana first learned about WAKIX^® (pitolisant) by doing their own research and was first interested in WAKIX because it’s not a stimulant. Tatiana was excited to talk to their healthcare provider to learn more about WAKIX and if it might be a good option for them. They asked their healthcare provider, and he explained possible side effects and set expectations for getting started on treatment. After Tatiana’s healthcare provider explained how to get started on treatment, including the titration period, and the importance of communication particularly during the first couple months, they started WAKIX in 2019.

“When I prescribe WAKIX to a patient, I make sure I set their expectations for getting started, including the titration period and that it might take up to 8 weeks for them to achieve a response,” Dr. Roy noted. “I start patients at a low dose and then may increase their dose each week until we get to the right dose for them.” Dr. Roy added, “WAKIX is thought to work differently through histamine levels in the brain.”

“It’s important that I inform my patients that WAKIX is not a stimulant and let them know that the way their body feels when taking WAKIX may be different from medications they’ve taken in the past,” Dr. Roy explained. “I also discuss the potential side effects, which, for adult patients, the most common are insomnia, nausea, and anxiety. I also mention other potential side effects and things they can expect while on treatment,” explained Dr. Roy. “I emphasize the importance of communicating with me as they are starting on treatment so we can discuss how they’re feeling, what improvements they’re seeing, and what side effects they might be experiencing. I also go over any questions they might have for me.”

Importance of support and community

Tatiana started to notice WAKIX was working when they had more wakefulness throughout the day and “weren’t falling asleep as uncontrollably as often.” Since they started taking WAKIX, Tatiana noticed that they were feeling less sleepy when riding in a car. “When I went on a road trip with my girlfriend, I was actually able to stay awake and have conversations with her,” shared Tatiana. They also felt less sleepy when doing activities they enjoy, like writing, reading, painting, and hiking. In August 2024, Tatiana had their poetry book titled “Wild Brujeria” published. In this book, Tatiana wrote about their life and has written multiple poems about living with narcolepsy.

Tatiana mentioned how the support of others in the narcolepsy community has also helped them through their journey and provided a source of hope and interpersonal support. “I show up and can help support people on my good days, and I can be supported on my bad days. Narcolepsy, it’s an up and down kind of thing…no one day looks the same. And it’s just having a community of people that understand and can support me and accept me, it’s been invaluable to my life, and I hope to pay it forward. Living with narcolepsy isn’t always easy. But for now, I’m thankful to have a treatment plan that is right for me,” Tatiana explained.

If you or someone you know is living with narcolepsy, speak with a healthcare provider to see if WAKIX could be an option. Read more about WAKIX below. To see videos about the experiences of Tatiana and others living with narcolepsy who are taking WAKIX, visit WAKIX Personal Stories.

Indications and Usage

WAKIX is a prescription medicine used to treat:
• excessive daytime sleepiness (EDS) or cataplexy in adults with narcolepsy.
• excessive daytime sleepiness (EDS) in children 6 years of age and older with narcolepsy.

Important Safety Information

Do not take WAKIX if you are allergic to pitolisant or any ingredient in WAKIX, or if you have severe liver disease.

WAKIX can cause a change in the electrical activity of the heart known as QT prolongation. This is a heart rhythm problem that can lead to an abnormal heartbeat. You have a higher chance of getting QT prolongation if you have certain heart or other medical conditions, or if you take WAKIX with certain medicines. Tell your healthcare provider right away if you have a change in your heartbeat or if you feel dizzy or faint while taking WAKIX.

Tell your healthcare provider about all your medical conditions, including if you have any heart, liver, or kidney problems, or problems with blood levels of your electrolytes, such as potassium or magnesium.

Tell your healthcare provider about all the medicines you take or plan to take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Taking WAKIX with certain medicines may cause side effects or affect the way each other works.

Hormonal birth control methods may not work while taking WAKIX. Use an alternative non-hormonal birth control method during treatment with WAKIX and for at least 21 days after stopping WAKIX.

Tell your healthcare provider if you are pregnant or planning to become pregnant. You are encouraged to enroll in the WAKIX pregnancy registry if you become pregnant while taking WAKIX. The registry collects information about the health of you and your baby. To enroll or obtain information from the registry,
call 1-800-833-7460.

The most common side effects of WAKIX in adults include insomnia, nausea, and anxiety. The most common side effects of WAKIX in children include headache and insomnia. These are not all the possible side effects of WAKIX. Call your healthcare provider for medical advice about side effects.

It is not known if WAKIX is safe and effective to treat excessive daytime sleepiness in children under 6 years of age with narcolepsy or to treat cataplexy in people under 18 years of age with narcolepsy.

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088. You can also report negative side effects to Harmony Biosciences at 1-800-833-7460.

Please see accompanying Full Prescribing Information.

2024-12-03T09:01:00

(BPT) – Every parent prioritizes the health and wellbeing of their child. For most, this means regular checkups and tracking their growth and development. But for some parents like Kim, it’s a different story, filled with hospital visits, multiple surgeries and advocacy for their child living with a rare disease.

When her son Quentin was around nine months old, Kim noticed marks on his torso and back, as well as visible tumors. By 12 months, a larger benign tumor had formed on the right side of his face, wrapping around his facial nerves.

“His cheeks were very full, like if you put two clementines on the side of his mouth and heavy and pulling down his right eye,” said Kim.

At 17 months, Quentin was diagnosed with neurofibromatosis type 1 (NF1), a rare genetic disorder that affects nearly 1 in 3,000 people worldwide. A lifelong and progressive condition, NF1 commonly appears in early childhood and can include spots on the skin, bone issues and other symptoms in the body. Then, at 22 months, Kim learned Quentin had plexiform neurofibromas (PN) — a type of tumor along the nerves of the body that develops in up to 50% of children with NF1. While PN tumors are noncancerous, they can still cause serious problems that impact a child’s quality of life.

“Because the tumor grew and wrapped around his facial nerve, it made it hard for Quentin to chew,” said Kim. “He couldn’t close his eyes, and his baby teeth were trapped inside the PN.”

Finding a management plan

Like many other children with NF1 PN, Quentin’s care was overseen by a team of specialists. For the first couple of years after his diagnosis, Quentin saw 14 physicians between two hospitals. “Our local hospital had a good handle on it, but the tumor was getting bigger, and we felt there was more we could try,” said Kim.

Over the next couple of years, Quentin underwent several surgeries to remove the PN on his face. Surgically removing PNs can be complicated and may not be an option for all patients. One reason is that the PN may be closely intertwined with critical nerves or blood vessels and located near major organs. Further, the PN may not be completely removed by surgery.

For Quentin, the surgeries were unsuccessful in removing the tumor from his face.

“He’s had 10 major surgeries, some that resulted in major complications,” said Kim. “They didn’t work. It was very difficult.”

When Quentin was five years old, one of his doctors determined he was eligible for a clinical trial evaluating a new treatment, selumetinib. His care team discussed with Kim the specific requirements for participation in the trial, including initial testing and continued record-keeping of Quentin’s symptoms, as well as the possible side effects of the treatment.

“Before the clinical trial, Quentin used to have around 20%-30% tumor growth every 3-6 months,” said Kim. “Once he started taking selumetinib, we noticed a significant shrinking of his tumor, and that the growth has since leveled off.”

This investigational treatment was approved by the FDA in April 2020 as KOSELUGO^® (selumetinib) which became the first and only prescription medicine for pediatric patients two years of age and older with NF1 who have symptomatic and inoperable PNs. KOSELUGO has been shown to reduce the size of some PNs, advancing care for children like Quentin who have inoperable PNs.

Quentin did experience some side effects, such as diarrhea, eczema and hair loss, but the progress was visible — his tumors were not growing, and Quentin has been taking KOSELUGO ever since.

The challenges and triumphs

Today, Quentin is 17 years old. He is still managing his NF1 PN, but thanks to his care team and support from his family and friends, he is a social and active teenager living an active life. Whether on the volleyball court or the baseball diamond, he is grateful for each opportunity.

“NF1 has shaped our lives positively and negatively,” said Kim. “Before we started therapy, he was in and out of the hospital and was septic at times. But on the positive side, NF1 made us closer, stronger and better communicators. We don’t take anything for granted.”

Quentin has been aware of his condition since he was four years old. Now, he unapologetically tells people he has NF1 PN and that there’s no cure. “Some days you’re swimming in the ocean, and some days asking for a life preserver,” reflected Kim.

For other parents and caregivers navigating their child’s NF1 PN diagnosis, Kim says that education and seeking resources are key.

“I can’t stress enough to advocate for your child and be educated yourself. Ask the questions. Ask, ask, ask,” she said. “Ask about support networks, what’s available in your community, and ask your doctors all the questions. You need to be educated in order to take care of someone else.”

This article is sponsored by Alexion, AstraZeneca Rare Disease.

IMPORTANT SAFETY INFORMATION

What are the possible side effects of Koselugo?

Koselugo may cause serious side effects, including:

• Heart problems. Koselugo can lower the amount of blood pumped by your heart, which is common and can also be severe. Your healthcare provider will do tests before and during treatment to check how well your heart is working. Tell your healthcare provider right away if you get any of the following signs or symptoms: persistent coughing or wheezing, shortness of breath, swelling of your ankles and feet, tiredness, increased heart rate.
• Eye problems. Koselugo can cause eye problems that can lead to blindness. Your healthcare provider will check your vision before and during treatment. Tell your healthcare provider right away if you get any of the following signs or symptoms: blurred vision, loss of vision, dark spots in your vision (floaters), other changes to your vision.
• Severe diarrhea. Diarrhea is common with Koselugo and can also be severe. Tell your healthcare provider right away the first time that you get diarrhea during treatment. Your healthcare provider may give you medicine to help control your diarrhea and may tell you to drink more fluids.
• Skin rash. Skin rashes are common with Koselugo and can also be severe. Tell your healthcare provider if you get any of the following signs or symptoms: rash that covers a large area of your body, peeling skin, blisters.
• Muscle problems (rhabdomyolysis). Muscle problems are common with Koselugo and can also be severe. Treatment with Koselugo may increase the level of a muscle enzyme in your blood called creatine phosphokinase (CPK) and may be a sign of muscle damage. Your healthcare provider should do a blood test to check your muscle enzyme levels of CPK before you start taking Koselugo and during treatment. Tell your healthcare provider right away if you get any of the following signs or symptoms: muscle aches or pain; muscle spasms and weakness; dark, reddish urine.

Before taking Koselugo, tell your healthcare provider about all your medical conditions, including if you:

• have heart problems.
• have eye problems.
• have liver problems.
• are pregnant or plan to become pregnant. Koselugo can harm your unborn baby. Your healthcare provider should verify if you/your partner are pregnant before beginning treatment. Ensure you/your partner use effective birth control (contraception) during treatment and for 1 week after your last dose if there is possibility pregnancy could occur. Tell your healthcare provider right away if you/your partner think you may be pregnant.
• are breastfeeding or plan to breastfeed. It is not known if Koselugo passes into your breast milk. Do not breastfeed during treatment and for 1 week after your last dose. Talk to your healthcare provider about the best way to feed your baby during this time.

Tell your healthcare provider about all the medicines you take, including prescription, over-the-counter medicines, vitamins, or herbal supplements. Especially tell your healthcare provider if you are taking aspirin, blood thinners, or other medicines to treat blood clots. Koselugo contains vitamin E, which may increase risk of bleeding.

What should I avoid while taking Koselugo?

Do not drink grapefruit juice, eat grapefruit, or take supplements with grapefruit or St. John’s Wort during treatment.

Most common side effects include: vomiting, stomach-area pain, nausea, dry skin, muscle and bone pain, feeling of tiredness or lacking energy, fever, sores in your mouth, headache, redness around the fingernails, itching.

These are not all the possible side effects of Koselugo. Call your healthcare provider for medical advice about side effects. Your healthcare provider may change your dose, temporarily stop, or permanently ask you to stop taking Koselugo if you have any of these side effects. You may report side effects to AstraZeneca at 1-800-236-9933 or at https://us-aereporting.astrazeneca.com or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

INDICATION

What is Koselugo?

Koselugo is a prescription medicine that is used to treat children 2 years of age and older with neurofibromatosis type 1 (NF1) who have plexiform neurofibromas that cannot be completely removed by surgery. It is not known if Koselugo is safe and effective in children under 2 years of age.

Please see Patient Information in the full Prescribing Information for Koselugo (selumetinib).

AstraZeneca and its Rare Disease Unit, Alexion, are committed to raising awareness of the disease and the need for treatment options. Learn more at Alexion.com and Astrazeneca-us.com. You can also visit Koselugo.com.

KOSELUGO is a registered trademark of the AstraZeneca group of companies. ALEXION is a registered trademark of Alexion Pharmaceuticals, Inc. © 2024, Alexion Pharmaceuticals, Inc. All rights reserved. All other trademarks are property of their respective owners.

US/KOS-NF1/0806 V1 07/2024