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The highly anticipated results come as the company makes significant changes to its C-suite. Despite the turnover, Dyne said it is looking toward expedited approval pathways for its DMD treatment.

The FDA has put a stop to U.S. initiation of PepGen’s Phase II trial for its Duchene muscular dystrophy treatment. The company faced the same hurdle for an earlier neuromuscular candidiate in 2023.

REGENXBIO Inc. (Nasdaq: RGNX) today announced it will participate in Cantor Fitzgerald’s Virtual DMD and Other Dystrophy Days on Wednesday, April 3, 2024.

Regenxbio is pushing its Duchenne muscular dystrophy gene therapy into pivotal development, with a BLA planned for 2026—potentially posing a threat to Sarepta’s Elevidys.

The investigational therapy, vesleteplirsen, had been positioned as an updated version of Sarepta’s original exon 51-skipping Duchenne muscular dystrophy drug Exondys 51.

Catalyst Pharmaceuticals, Inc. today announced the U.S. commercial launch of AGAMREE® (vamorolone) oral suspension 40 mg/mL for the treatment of Duchenne Muscular Dystrophy (“DMD”) in patients aged two years and older.

We are in an unprecedented time in neurotherapeutics. Medicines that address the causative disease biology underlying central nervous system

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced U.S. Food and Drug Administration (FDA) approval of an expansion to the labeled indication for ELEVIDYS (delandistrogene moxeparvovec-rokl) to include individuals with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene who are at least 4 years of age.