Business/Regulatory

In this exclusive interview with RDR, Dr. Francois Nader discusses the history of NPS Pharmaceuticals.
“The Committee accepted that eculizumab is a step change in the management of aHUS and can be considered a significant innovation for a disease with a high unmet clinical need.”
BioMarin Pharmaceuticals announced it plans to buy Prosensa Holding NV for approximately $840 million ($17.75 for all shares).
As they say, “we are now open for business”
The open-label study will evaluate the efficacy and safety of eteplirsen in Duchenne muscular dystrophy patients over 48 weeks (30 mg/kg once a week)and in approximately 39 sites across the United States.
I never imagined I would be the CEO of a small rare disease company.
Evaluate Pharma has released their report on orphan drugs and what the landscape will look like in 6 years.
AstraZeneca is joining up with Pharmacyclics and Janssen to hopefully improve clinical outcomes in a number of hematologic cancers.
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