Patients, Supporters Worldwide Recognizing Rare Disease Day 2020
An abundance of events are afoot around the world to mark Rare Disease Day 2020 on Feb. 29. The activities are focused on heightening ... Read more
Updates
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Antimalarial Medicine Chloroquine Improves Muscle Strength in Animal Models of DM1, Study Finds
Treatment with chloroquine increased the levels and restored the activity of Muscleblind-like RNA-binding proteins, improving muscle function and strength in animal models of ... Read more -
PTC Therapeutics’ Global Program Strives to Fund Innovative Projects of Patient Advocacy Organizations
PTC Therapeutics has launched its sixth annual global grant program for patient advocacy organizations that seek to develop innovative projects for the Duchenne muscular ... Read more -
MDA Launches 38th Annual #Shamrocks Fundraising Campaign, Aims to Raise $8M
The Muscular Dystrophy Association (MDA) has launched its 38th annual #MDAShamrocks campaign — the largest St. Patrick’s fundraiser in the U.S. — aiming to ... Read more -
Duchenne ‘Consumer Advocates’ Have Say in Federal Research Funding for DMD
People known as “consumer advocates” have joined top scientists since 2011 in helping to determine how funds appropriated by the U.S. Congress will be ... Read more
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Patients, Supporters Worldwide Recognizing Rare Disease Day 2020
An abundance of events are afoot around the world to mark Rare Disease Day ... -
As Adults with DMD, We Have a Responsibility to Mentor Younger Generations
Adults with Duchenne muscular dystrophy (DMD) should help younger generations through the difficult parts ... -
Registration for PPMD Advocacy Conference Now Open
Registration for the upcoming Parent Project Muscular Dystrophy (PPMD) Advocacy Conference is now open. ... -
TAS‐205 Safe, May Slow Muscle Loss in DMD Patients, Phase 2 Data Suggest
Oral treatment with TAS-205 is safe, and may delay muscle deterioration in patients with ... -
Addressing the Funding Crisis for Social Security
For those of you who follow my column, I have some good news: Sammy ... -
CureDuchenne Teams Up with Vivli to Share DMD Clinical Trial Data
Seeking to improve treatment of Duchenne muscular dystrophy (DMD), CureDuchenne has teamed up with Vivli ...
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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