Healio20d
FDA grants fast-track designation to myotonic dystrophy type 1 treatment
Please provide your email address to receive an email when new articles are posted on . The FDA has granted fast-track designation to an investigational antisense oligonucleotide to treat ...
Gilbert Gottfried’s Final Comedy Album Is Helping Explore Treatment for the Rare Condition That Led to His Death
The comic's widow, Dara Gottfried, says a portion of sales of 'Still Screaming' will benefit the Gilbert Gottfried Myotonic ...
WebMD27d
Understanding Muscular Dystrophy: Symptoms
Other symptoms may include: Often children with Becker muscular dystrophy can walk. As they get older they may need to use a cane or wheelchair to get around. The symptoms of myotonic dystrophy ...
Business Insider21d
Dyne Therapeutics Receives FDA Fast Track Designation for DYNE-101 for the Treatment of Myotonic Dystrophy Type 1
About Myotonic Dystrophy Type 1 (DM1) DM1 is a rare, progressive, genetic disease that affects skeletal, cardiac and smooth muscle. It is a monogenic, autosomal dominant disease caused by an ...
Arrowhead Pharmaceuticals Announces Closing of Global License and Collaboration Agreement with Sarepta Therapeutics
Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) today announced that the global licensing and collaboration agreement with Sarepta Therapeutics (NASDAQ: SRPT) announced on November 26, 2024, has now ...
Morningstar21d
Dyne Gets FDA Fast-Track Status for Neuromuscular Product Candidate
Dyne Therapeutics received fast-track designation from the Food and Drug Administration for a treatment of myotonic dystrophy type 1, a neuromuscular disease. The Waltham, Mass., developer of ...
Yahoo Finance17d
Dyne Therapeutics Receives FDA Fast Track Designation for DYNE-101 for the Treatment of Myotonic Dystrophy Type 1
has granted Fast Track designation for DYNE-101 for the treatment of myotonic dystrophy type 1 (DM1). DYNE-101 is currently being evaluated in the ongoing Phase 1/2 ACHIEVE global clinical trial.