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
Updates
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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 suggest its effectiveness as a treatment for facioscapulohumeral muscular ... Read more -
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 in Cleveland, Ohio, has been postponed until July 18–20 because ... Read more -
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 Exondys 51 (eteplirsen), both approved for the treatment ... Read more -
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), will not be affected by the COVID-19 pandemic, ... Read more
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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 ... -
Machine Learning Helps Diagnose Specific MD Forms, Study Finds
Machine learning could be used to help diagnose specific forms of muscular dystrophy (MD) ... -
Capricor Therapeutics Expands Exosome Technology for DMD
Capricor Therapeutics is expanding the development of exosome-based technologies that are being tested as ... -
Redefining Success While Living with a Chronic Illness
I never have an answer to the questions, “Where do you see yourself in ... -
AdipoRon Eases Inflammation, Improves Motor Function in DMD Mice, Study Shows
Oral treatment with AdipoRon reduced inflammation in muscle, promoted muscle fiber proliferation, and improved ...
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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