Keyword: Sarepta

One of the first six people to receive the treatment was hospitalized with acute renal injury, leading analysts to talk down Pfizer’s chances.

Sarepta licensed five gene therapies for limb-girdle muscular dystrophy from Myonexus last May. Now it's buying Myonexus outright for $165 million.

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.

After refocusing on gene therapy, Axovant is appointing five new executives to posts ranging from clinical development to commercial operations.

Preliminary signs of improved muscle function have been seen in Sarepta's Duchenne muscular dystrophy gene therapy trial.

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.

The initiative taps into the networks Invitae has built up to connect sponsors to patients and the clinicians responsible for their care.

Sarepta licenses five new gene therapies to its muscular dystrophy portfolio from Myonexus—and takes an option on buying the company.

Buoyed by safety data and evidence of exon-skipping activity, Daiichi is pushing ahead with further development of the antisense oligonucleotide.