ALL
Pharma in China
Pharma Experts
Market News
Products Guide
Brand Story
-
Aruvant Receives FDA Rare Pediatric Disease Designation for Sickle Cell Treatment
January 13, 2020Aruvant announced the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease designation to ARU-1801, Aruvant's investigational therapy for the treatment of sickle cell disease. -
Orchard Therapeutics’ OTL-200 Receives Rare Pediatric Disease Designation from FDA for Treatment of Metachromatic Leukodystrophy
May 08, 2018Orchard Therapeutics, a leading commercial stage company dedicated to transforming the lives of patients with rare diseases through innovative gene therapies...
Pharma Sources Insight January 2025
