Topic: Duchenne muscular dystrophy (DMD)
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.
Catabasis is looking to test its experimental Duchenne drug edasalonexent in a late-stage trial.
A panel of experts convened by the FDA has overwhelmingly knocked back PTC Therapeutics’ filing for approval of ataluren.
Wednesday's shot of your news of note.
Shares in the biotech shot up 60% after it posted phase 2 data that suggest it can muscle in on a market fought over by Boehringer and Roche.
A University of Missouri-led team developed a new gene transfer method to treat Duchenne muscular dystrophy.
University of Utah scientists found antisense drugs reversed symptoms in mouse models of ataxia and amyotrophic lateral sclerosis.