Rare Disease
Expertise: Rare Disease 78 results
In rare diseases, patient opinion and insight often go unheard. However, for sponsors of rare disease clinical trials, study success often rests on incorporating the patient perspective as early as possible in development. Many patients with rare diseases have undertaken...
Rare Disease
Jackie Brown
Rare Disease
Why Rare Disease Day Matters to Us
An estimated 300 million people globally suffer from a rare disease. Further still, 25 million people in the U.S. alone are living with an undiagnosed rare disease. Limitations in disease understanding and lack of approved treatments can mean countless challenges...
Developing a novel gene therapy requires a level of comfort with change and unpredictability. The path to regulatory approval is rarely straightforward, and roadblocks can enter at any turn. Premier Research was approached by a sponsor of an adeno-associated virus...