What’s Next for Innovation in Pediatric Rare Disease Treatments?
For children battling rare and often life-threatening diseases, access to new therapies can be a lifeline. Yet, developing drugs for […]
orphan drug designation FDA
Navigating Clinical Development Challenges for Rare Disease Therapies
Rare diseases, as defined by the Orphan Drug Act (ODA), affect fewer than 200,000 people in the United States. Although