Business/Regulatory

Kevin Lee, PhD, chief scientific officer of Pfizer's rare disease research unit discusses Pfizer's philosophy on orphan drug development.
Rare Disease Community is a collaborative one between patients and industry. In this exclusive interview with Rare Disease Report, Kevin Lee, PhD, chief scientific officer of Pfizer's rare disease research unit discusses some of the programs Pfizer has established to help the drug developers learn what the patients want and need.
2014 was a difficult year for Sarepta.
Shire has bought Meritage, a privately-held company for their oral budesonide suspension that is in phase 3 development.
Two companies were recently given 10-year National Institute of Neurological Disorders and Stroke (NINDS) awards from the National Institutes of Health (NIH) to advance drug development and provide manufacturing services for potential treatments for neurological disorders.
Rare Disease Report had the opportunity to speak with Todd C. Brady, MD, PhD, President and CEO of Aldeyra Therapeutics, about NS2 for the treatment of Sjögren-Larsson Syndrome.
Genzyme is growing its the neurology business, thanks to collaboration with Voyager Therapeutics. The two companies plan to collaborate to develop and market novel gene therapies for severe central nervous system (CNS) disorders, such as Huntington’s disease, Friedreich’s ataxia, and Parkinson’s disease.
Starting, running, or even working in a pharmaceutical company focused on orphan drugs requires people to think differently.
Shire plc and NPS Pharma have entered into a merger agreement in which Shire will acquire all the outstanding shares of NPS Pharma for approximately $5.2 billion ($46.00 per share in cash). The transaction has been approved unanimously by the Boards of Directors of both Shire and NPS Pharma.
The NPS in NPS Pharma stands for Nexus of Patients and Science.
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