Topic: Duchenne muscular dystrophy (DMD)
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.
The data, taken from three patients, showed a 38% increase in a muscle protein that boys with DMD usually lack.
The FDA lifted the second of two clinical holds on Solid Bio’s Duchenne muscular dystrophy trial, following safety and manufacturing concerns.
At BIO, we chatted with Doug Ingram about Sarepta's pipeline, its latest hires and how the healthcare landscape must change.
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.