The ongoing Phase 2 HOPE-2 study has been cleared to continue exploring the potential of CAP-1002 therapy in boys and young men with advanced Duchenne muscular dystrophy (DMD). An independent Data and Safety Monitoring Board (DSMB) made the decision after its thorough safety and futility review and the…
Screening newborns for genetic diseases with treatments that can prevent crippling or deadly progression, especially for rare disorders, has a ways to go in the United States. No state today tests for all 35 disorders recommended under a federal screening panel, and even in those that come close, rare…
Climbing up the world’s tallest mountain isn’t for everyone. With that in mind, a small New England-based charity plans just such an adventure this November — for the third time, no less — to raise at least $100,000 to help cure Duchenne muscular dystrophy (DMD). Tonya Dreher is executive…
Invitae has opened a program offering free genetic testing and post-test counseling to people suspected of having diseases that include muscular dystrophy (MD) under a partnership with pharmaceutical firms. The goal is to promote early diagnosis and treatment. The initiative, called Detect, targets disorders for which testing…
Filippo Buccella, an Italian pharmacist from Rome, clearly remembers the day in 1993 when he brought his nearly 3-year-old son, Luca, to the hospital. Luca was about to undergo surgery for his adenoids when a test revealed that his creatine kinase level was over 11,000 — a hallmark symptom of…
If there’s anyone who knows a thing or two about how to discuss Duchenne muscular dystrophy (DMD) with your son, his siblings, his teachers and his friends, it’s James Poysky. A clinical psychologist in Katy, Texas, Poysky teaches at Houston’s Baylor College of Medicine and chairs a…
Back in June 1984, a doctor calmly informed Pat Furlong that her sons — 4-year-old Chris and 6-year-old Patrick — had Duchenne muscular dystrophy. It was the first time she had ever heard those three words uttered together. The disease eventually claimed both boys, but that didn’t stop Furlong’s fight against…
The Muscular Dystrophy Association (MDA) awarded $389,000 in funding to a researcher from University of California, Los Angeles (UCLA) to support the development of a small molecule that holds promise for preventing muscle damage in Duchenne muscular dystrophy (DMD). The molecule raises levels of a…
A new international consortium based in Paris, and funded largely by the 28-member European Union, intends to speed the diagnosis of rare diseases, while also accelerating the development of treatments for the 95% of such illnesses that currently don’t have one. The European Joint Programme on Rare Diseases (EJP…
CAP-1002, Capricor Therapeutics’ investigational cell therapy for Duchenne muscular dystrophy (DMD), significantly improved skeletal muscle and lung function in a group of boys and young men at advanced stages of the disease, top-line data from an interim analysis of a Phase 2 trial show. CAP-1002…
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