First Hint of What’s to Come With Sarepta and BioMarin

Insights - The event of the week - some would argue the event of the year - was the release of FDA briefing documents related to BioMarin's (BMRN) drisapersen, which faces an FDA advisory committee meeting this coming Tuesday. The drug is up for approval in Duchenne Muscular Dystrophy next month, and the upcoming committee meeting is all about FDA getting input from outside experts on the risk/benefit of approving drisapersen for DMD patients amenable to treatment by exon 51 skipping, drisapersen's mechanism of action.

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Filling the Void in Beta Thalassemia

Research - Bellicum Pharmaceuticals (BLCM) after the bell this evening reported third quarter earnings, but more importantly noted that four pediatric beta-thalassemia patients who underwent HSCT followed by BPX-501 modified T cells are transfusion-free.