Morning Briefing
Summaries of health policy coverage from major news organizations
Jaw-Dropping Results From Experimental Duchenne Muscular Dystrophy Treatment Sparks Hope In Patients
Stat: Sarepta’s Gene Therapy For Duchenne Raises Hopes For ‘Real Change’
An experimental gene therapy for Duchenne muscular dystrophy, licensed to Sarepta Therapeutics, produced jaw-dropping increases in a crucial muscle protein normally missing in patients with the disease, according to preliminary clinical trial data released Tuesday. The data were collected from just three boys, but the effect of the gene therapy — producing 38 percent of a truncated form of the normal dystrophin muscle protein — is profound enough to suggest it may halt or even reverse the effects of Duchenne in certain patients. (Feuerstein, 6/19)
The Wall Street Journal: Sarepta Gets Boost From Early Trial Results On Muscular Dystrophy Treatment
Sarepta said study results showed the therapy helped patients with the crippling disease significantly reduce levels of creatine kinase, an enzyme associated with muscle damage caused by the disorder. Results also showed patients receiving the treatment had robust levels of a protein key to muscle function. No serious adverse events were observed in the study, the Cambridge, Mass., biopharmaceutical company said. (Al-Muslim, 6/19)
Stat: What Sarepta's Game-Changing Data Mean For Other Duchenne Players
Sarepta Therapeutics became the toast of biotech on Tuesday after revealing early data on a gene therapy for Duchenne muscular dystrophy that far outstripped Wall Street’s expectations. But Sarepta is hardly alone in the field, and nothing in biotech happens in a vacuum. Here’s a look at what the latest DMD results mean for the company’s competitors. (Garde, 6/19)
