Topic: Duchenne muscular dystrophy (DMD)
The FDA has extended its partial hold on Solid Bio's phase 1/2 DMD trial to a full hold following a patient's hospitalization.
The latest analysis links the utrophin modulator to a statistically significant decline in muscle inflammation in boys with DMD.
Sarepta expects to resume dosing in a U.K. trial of its DMD drug golodirsen pending MHRA approval.
Targeting "clusters" of mutations with CRISPR could lead to a treatment that could work for up to 60% of patients with DMD.
Solid Bio has pressed on after its plans for an IPO fell apart—with a cut-price offering for an upsized stake.
At 24 weeks, Summit's ezutromid raised utrophin levels and reduced muscle damage in a phase 2 trial.
We catch up with Doug Ingram, chief at rare disease biotech Sarepta, and discuss how he aims to remove the term “controversial” in front of the company’s name.
The takeover gives Astellas full control of a phase 1 DMD drug in return for $225 million upfront and the same again down the line.
Sarepta has been signing a series of new R&D pacts since its controversial DMD drug approval last fall, and today it’s penned a deal for one of the hottest research areas around: CRISPR.
UC Berkeley scientists are using a new CRISPR delivery system to more safely correct the mutated dystrophin gene in mouse models of DMD.