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.

They help doctors identify diseases that affect the nerves and muscles as well as the extent of any damage. This test is used to diagnose myotonic muscular dystrophy, which causes muscles to stay ...

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 ...

PGN-EDODM1 is under clinical development by Pepgen and currently in Phase I for Myotonic Dystrophy. According to GlobalData, Phase I drugs for Myotonic Dystrophy does not have sufficient historical ...

The investigational therapy DYNE-101 worked as expected and led to functional improvements for people with myotonic dystrophy type 1 (DM1) in a clinical trial, according to an update from developer ...

There is a simple discount patient access scheme for vamorolone. NHS organisations can get details on the Commercial Access and Pricing (CAP) Portal. Non-NHS organisations can contact ...

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 ...