Business/Regulatory

Each year, over 75,000 hospitalizations occur in the United States as a result of vaso-occlusive criss. Andyjr average hospital stay for such a crisis is 6 days.
The review process for drisapersen is not only being watched closely by the Duchenne community, but also by investors of Biomarin and Sarepta.
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).
Carcinoid syndrome is a rare condition that often is often difficult to diagnose and manage. A key person in the management of patients with carcinoid syndrome is the oncology nurse.
Pablo Lapuerta, MD, chief medical officer at Lexicon Pharmaceuticals discusses the pathophysiology of carcinoid syndrome, a rare condition that can take years to properly diagnose.
April is Fabry Disease Awareness Month. Fabry disease is an inherited lysosomal disorder that primarily affects males.
Currently, no FDA-approved treatments are available for rare mitochondrial myopathies.
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.
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