The long-awaited decision by the European committee CHMP was announced recently by Amylyx Pharmaceutical (the Company) based in Cambridge, Massachusetts just as anticipated. The EMA issued its negative opinion of…
Earlier this year, the FDA granted Accelerated Approval to ELEVIDYS (delandistrogene moxeparvovec-rokl) for the treatmnt of Duchenne muscular dystrophy (DMD) in boys aged 4-5. While this is a stunning…
If gastric cancer is localized (has not yet spread), it carries a significantly higher 5-year survival rate of 72-75%. But once the cancer spreads, the rate falls lower: 35%…
NOTE: This story was provided through the CureGRIN Foundation, a Patient Worthy partner organization. Written by Lauren Williams Why Me, Why Not A question I struggled terribly with at the…
People in China who are living with narcolepsy will soon have access to a new therapeutic option to help them manage cataplexy or excessive daytime sleepiness (EDS) after the…
Patient Worthy partner Chelsea's Hope is a nonprofit organization dedicated to supporting families impacted by the deadly rare illness Lafora disease, as well as amplifying research. Recently, a documentary titled Fighting…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
The 25th World Congress of Dermatology took place this year from July 3 to 8, 2023. During the Congress, stakeholders throughout the medical industry convened to discuss research, trends, and…
According to a story from SciTech Daily, a team of researchers affiliated with the University of Zurich's ITINERARE University Research Priority Program recently collaborated with Insilico Medicine to conduct research…
Two days. That’s all it took for Jill and Jeff Ackermann to watch their son’s condition mysteriously deteriorate. On Saturday, six-year-old Bryson was the energetic, athletic, and bright-eyed little boy…
Having a rare or uncommon condition can be undoubtedly overwhelming. The diagnosis comes with associated mental health, financial, physical, and social burdens that can weigh heavy on your mind. But…
According to a story from Vox, Americans are some of the world's most voracious meat eaters. Per capita, Americans eat an average of 60 lb. of red meat each year,…
Note: These quotes and reflections are from members of the cystinosis community who were in attendance at the 2023 Cystinosis Research Network Family Conference which took place in Nashville, TN…
In 2020, the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy designation to magrolimab for the treatment of newly diagnosed myelodysplastic syndromes (MDS). This designation is designed to expedite…
According to a story from Market Screener, the drug company AbbVie recently announced that its investigational therapy epcoritamab has received a positive opinion from the European Union's Committee for Medicinal…
There are an estimated 10,000 rare diseases. A majority of rare diseases are genetically oriented. Because of this, we learn more about health and disease as we learn more…
According to a story from WGN-TV Chicago, treatment for esophageal cancer can be grueling on patients, even if successful. Surgery is often a critical aspect of the process and can…
Rare Community Profiles Rare Community Profiles is a new Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their…
Professor Timothy Yu developed the custom drug milasen named in honor of Mila, an 8-year-old girl with Batten disease. The drug is the first drug specifically designed for one…
Lonnie's mother, Stephanie Pesterfield of Colorado Springs, still finds it difficult to talk about her son Lonnie’s near-death experience. Yet as published recently in People, Stephanie and her son want…
Growing up, Jack Witzig saw how his father inspired others. His dad Don was a cross country and track coach at two different high schools. He motivated his students to…
Family gatherings can be fun, boisterous, and sometimes full of conflict. During one such event, Donnie Adams found himself intervening to break up a fight between two people. Although he…
When Chantel and Jason Funk learned that their son Bowin has adrenoleukodystrophy (ALD) in 2019, they thought that the questions about the future, and how to manage the condition,…
Marco and India Biviano felt like something bad was happening to their young son Luca. His stomach was distended and painful. Luca frequently developed chest infections that required round after…
Leukemia is cancer that begins in the bone marrow and involves abnormal white blood cells (WCB) from lymphatic tissues and bone marrow. Scientists have new theories as to why certain…
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