The US Food and Drug Administration (FDA) has approved the antisense oligonucleotide casimersen (Amondys 45, Sarepta Therapeutics) injection for the treatment of patients with Duchenne muscular ...

DMD gene therapy faces challenges in safety, efficacy, and long-term outcomes, despite FDA approval of delandistrogene moxeparvovec. Future strategies focus on novel AAV capsids, dual/triple vector ...

Duchenne muscular dystrophy (DMD) is a rare neurological condition that causes severe muscle weakness and intellectual ...

Duchenne muscular dystrophy (DMD) is a genetic disease that causes progressive muscle weakness. DMD commonly affects boys, where symptoms are seen in early childhood. There is no cure for DMD. Health ...

TAS-205, targeting hematopoietic prostaglandin D synthase, showed no significant efficacy in improving time to rise in DMD patients over 52 weeks. The REACH-DMD trial was a placebo-controlled, ...

Muscular dystrophy is a disease caused by mutations in the dystrophin gene. The muscles of muscular dystrophy patients weaken and atrophy over time. Heart and breathing muscles may also eventually be ...

Upgrading Dyne Therapeutics to 'Buy' due to promising late-stage DM1 and DMD drug candidates and significant unmet need in both indications. Recent management changes and a more measured clinical ...

The European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended renewal of conditional marketing authorization for ataluren (Translarna, PTC Therapeutics) for ...

Using a new gene-editing technique, a team of scientists from UT Southwestern Medical Center stopped progression of Duchenne muscular dystrophy (DMD) in young mice. If efficiently and safely scaled up ...