candidate for myotonic dystrophy type 1 (DM1), and del-brax for facioscapulohumeral muscular dystrophy (FSHD). All three candidates are based on the same TfR1-targeting antibody component.

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 ...

Muscular dystrophy is caused by defects in certain genes, with type determined by the abnormal gene. In 1986, researchers discovered the gene that, when defective or flawed, causes Duchenne ...

There are several types of muscular dystrophy. Muscle weakness is a hallmark of each type. But the symptoms can vary and start at different ages. Some muscular dystrophies are mild. Others are ...

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.

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 ...

Entrada Therapeutics plans to initiate ELEVATE-44-201 trial for ENTR-601-44 in Duchenne muscular dystrophy ... for myotonic dystrophy type 1. This press release contains express and implied ...