Self-quarantine Has Leveled the Social Playing Field
I’ve always sought silver linings, including in this season of social distancing. Many people now exclusively use technology to communicate with family, friends, and ... Read more
Updates
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NORD’s Advice on COVID-19: You’re Stronger Than You Think
First, the bad news: If you’re one of the 30 million or so Americans with a rare disease, you probably have lower immunity to ... Read more -
Trial Use of Edasalonexent Not Likely to Raise COVID-19 Risk, Catabasis Reports
Clinical trial data suggest that edasalonexent — a potential Duchenne muscular dystrophy (DMD) therapy — does not affect the immune system’s ability to work as ... Read more -
The Muscular Dystrophy Community Counts on Accessibility Improvements
I feel strange not writing about the pandemic crisis in this column, but we’re already bombarded with information about it, and it appears we ... Read more
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Phase 1 Results Support Losmapimod as FSHD Treatment
Final results of a Phase 1 clinical trial support the safety of losmapimod and ... -
NORD Partners with ‘Sing Me a Story’ for July 18–20 Living Rare Forum
The Living Rare, Living Stronger Patient and Family Forum, originally set for May 14–16 ... -
Sarepta Working to Ensure Treatment Access, Clinical Trials Continuity Amid COVID-19 Pandemic
Sarepta Therapeutics has taken steps to ensure the supply of Vyondys 53 (golodirsen) and ... -
Emflaza Supply Not Affected by COVID-19 Pandemic, PTC Therapeutics Says
The supply of Emflaza (deflazacort), a treatment for people with Duchenne muscular dystrophy (DMD), ... -
Global Rare Disease Group’s Focus: 1,000 New Therapies by 2027, Despite COVID-19
Even with the coronavirus pandemic ravaging Europe and much of the world, patient advocate ...
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FDA Grants Rare Pediatric Disease Status to Myonexus’ MYO-101 for LGMD 2E
News
The U.S. Food and Drug Administration recently granted rare pediatric disease designation to Myonexus Therapeutics’ experimental gene therapy MYO-101 for the treatment of limb girdle muscular dystrophy (LGMD) type 2E, or LGMD2E. The announcement was made by Myonexus and Nationwide ... Read more