Duchenne Muscular Dystrophy

Stealth BioTherapeutics Corp (NASDAQ: MITO) presented new data demonstrating the potential synergistic relationship between elamipretide and an exon-skipping phosphorodiamidate morpholino oligomer (PMO) in the X-linked muscular dystrophy (mdx) animal model.  The data were...

The FDA has placed a clinical hold on Dyne Therapeutics Inc's (NASDAQ: DYN) investigational New Drug (IND) application to initiate a clinical trial of DYNE-251 in patients with Duchenne muscular dystrophy (DMD) amenable to skipping exon 51.  Dyne received a clinical...

In a community letter, Pfizer Inc (NYSE: PFE) informed the Parent Project Muscular Dystrophy of the death of a young man in the Phase Ib trial of its mini-dystrophin gene therapy. The event has triggered a halt in screening and dosing and a clinical hold imposed by the FDA. Pfizer wrote...

Edgewise Therapeutics Inc (NASDAQ: EWTX) has announced topline results from the MAD portion of Phase 1 trial of EDG-5506, designed to protect skeletal muscle fibers in Duchenne muscular dystrophy (DMD) and Becker Muscular Dystrophy (BMD). EDG-5506 was shown to be generally well-...

Sarepta Therapeutics Inc (NASDAQ: SRPT) shared new analyses and functional data from its SRP-9001 development program and the details of Study SRP-9001-301 (EMBARK) Phase 3 trial of SRP-9001 for Duchenne muscular dystrophy.  SRP-9001, being developed in partnership with ...

Dyne Therapeutics Inc (NASDAQ: DYN) has announced new data presented today during the 2021 Muscle Study Group Annual Scientific Meeting for its Duchenne muscular dystrophy (DMD) program. DMD is a rare disease caused by mutations in the gene that encodes for dystrophin, a protein...

Capricor Therapeutics Inc (NASDAQ: CAPR) has announced final data from Phase 2 HOPE-2 trial of CAP-1002 for advanced stages of Duchenne muscular dystrophy (DMD).  The trial met its primary efficacy endpoint of mid-level performance of upper limb (mid-PUL v1.2) and various...

Solid Biosciences Inc (NASDAQ: SLDB) reports 1.5-year functional data and patient-reported outcome measures in the ongoing IGNITE DMD Phase 1/2 trial of SGT-001.  SGT-001 is a novel adeno-associated viral (AAV) vector-mediated gene transfer therapy for Duchenne muscular...

Sarepta Therapeutics Inc (NASDAQ: SRPT) has announced positive 12-week results from the first 11 participants enrolled in open-label study 9001-103 ENDEAVOR being conducted in partnership with Roche Holdings AG's (OTC: RHHBY). Results demonstrated robust expression of...

Sarepta Therapeutics Inc (NASDAQ: SRPT) announced positive results from Part A in a tiny group of 4 patients of the MOMENTUM Phase 2 study evaluating SRP-5051 in Duchenne muscular dystrophy (DMD) patients amenable to exon 51 skipping. SRP-5051 is the company's...

Sarepta Therapeutics Inc (NASDAQ: SRPT) has entered a partnership with Roche (OTC: RHHBY) to commercialize SRP-9001, its gene therapy for Duchenne Muscular Dystrophy, outside of the U.S. The deal secures Sarepta a $1.15 billion upfront payment — $750 million in cash and $400 million in an...

Roche Holdings AG Basel ADR (OTC: RHHBY) said Monday that it has signed a licensing agreement with Sarepta Therapeutics Inc (NASDAQ: SRPT) to acquire the commercial rights to the latter's investigational gene therapy asset SRP-9001 outside of the U.S. The asset is being evaluated in...

Solid Biosciences Inc (NASDAQ: SLDB) shares are advancing strongly Wednesday following on an update on its clinical development program for its SGT-001, its gene transfer investigational candidate for Duchenne muscular dystrophy, or DMD. Biomarker Data Encouraging Solid Biosciences said biomarker...

After an earlier rejection, Sarepta Therapeutics Inc (NASDAQ: SRPT)'s Vyondys 53 received FDA approval the second time around, sending the stock sharply higher Friday. Sarepta Succeeds On Second Try  Cambridge, Massachusetts-based Sarepta said Thursday that the FDA granted...

Solid Biosciences Inc (NASDAQ: SLDB) shares pulled back to all-time lows Tuesday after the company said its lead gene therapy candidate to treat Duchenne muscular dystrophy has been placed on clinical hold by the FDA yet again. FDA Seeks Study Suspension On Safety Scare Solid Biosciences...