Rare Disease Report spoke with Dr. Seng Cheng, PhD, Head of Research and Early Development, Rare Diseases Division at Genzyme about this collaboration and what it could eventually mean for the cystic fibrosis community.
After a long, hard battle with the FDA, Sarepta Therapeutics is now ready to submit a rolling New Drug Application (NDA) for eteplirsen to treat boys with Duchenne muscular dystrophy (DMD) amenable to exon-51 skipping therapy (about 13% of DMD boys).
Sarepta Therapeutics has announced that Chris Garabedian has resigned as President, Chief Executive Officer (CEO), and as a member of the Board of the company, effective immediately.
Edward Kaye, MD, the company’s Chief Medical Officer (CMO) will act as interim CEO.
Patient perspectives generate specific outcome objectives, what they are willing to do to achieve them, the risks they are willing to bear, and the costs they are willing to pay. The same could be said about payers.