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

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

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.

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.

On the heels of a Phase III flop for Pfizer’s Duchenne muscular dystrophy gene therapy candidate, the FDA has green lighted the expanded use of Sarepta Therapeutics’ Elevidys.

Capricor Therapeutics today announced that the U.S. Food and Drug Administration (FDA) has scheduled a Pre-BLA (Biologics License Application) meeting with the Company in the third quarter of 2024 for deramiocel (CAP-1002), for the treatment of Duchenne Muscular Dystrophy (DMD).

Dyne Therapeutics, Inc. announced positive clinical data from its ongoing Phase 1/2 ACHIEVE trial of DYNE-101 in patients with myotonic dystrophy type 1 and its ongoing Phase 1/2 DELIVER trial of DYNE-251 in patients with Duchenne muscular dystrophy who are amenable to exon 51 skipping.

While Thursday’s label expansion and traditional approval for the gene therapy is an important milestone, many challenges still face the Duchenne muscular dystrophy community.

Italfarmaco S.p.A. announced today that the full results from the Company’s pivotal phase 3 EPIDYS clinical trial with givinostat in ambulant boys 6 years of age and older with Duchenne muscular dystrophy (DMD) have been published in The Lancet Neurology.

As Sarepta Therapeutics moves closer to full approval and an expanded label for its gene therapy, some experts push back on clinical efficacy and cost while others note the hope it provides patients with Duchenne muscular dystrophy.