Expertise: Rare Disease 67 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 a long journey to diagnosis, so participating in a study may feel like a huge…
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 and diminished quality of life for many battling a rare disease. For all those looking…