Topic: Duchenne muscular dystrophy (DMD)
A Duke lab at the center of a research pact with Sarepta is reporting promising results from a trial of CRISPR gene editing in mouse models of DMD.
Solid Bio’s run of negative news is continuing with another setback for its Duchenne muscular dystrophy gene therapy.
Santhera plans $50 million placement to fund the purchase of a license to ReveraGen’s Duchene muscular dystrophy drug vamorolone from Idorsia.
Preliminary signs of improved muscle function have been seen in Sarepta's Duchenne muscular dystrophy gene therapy trial.
The early-stage alliance comes a week after Pfizer halted development of domagrozumab in DMD because of lackluster midphase data.
Using CRISPR to correct a DMD gene mutation restored up to 92% of muscle function in dogs.
Another gene therapy alliance shows that Sarepta won’t allow an issue with its lead gene therapy candidate to dampen its enthusiasm for the field.
A trial of Sarepta’s gene therapy for muscular dystrophy was placed on clinical hold by the FDA but could be upgraded to a pivotal study as a result.
Two AI firms, Insilico Medicine and A2A Pharmaceuticals, launched a joint company aimed at Duchenne muscular dystrophy and other rare orphan diseases.
Despite posting promising early data, Summit Therapeutics' Duchenne muscular dystrophy drug missed its primary and secondary endpoints in a phase 2 trial.