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Pharmaceutical Technology1d
Overcoming challenges in orphan drug development: From clinical trials to market
Developing orphan drugs involves navigating intricate regulatory landscapes, financial risks, and limited clinical data. To ...
Shuttle Pharma FDA Orphan-Drug Nears Patient Enrollment Milestone for Phase 2 Clinical Trial
Shuttle Pharmaceuticals Holdings, Inc. (Nasdaq: SHPH) (“Shuttle Pharma” or the “Company”), a discovery and development stage ...
FDA-designated orphan drug could increase radiation efficacy in lung cancer
An FDA-designated orphan drug that can target a key vulnerability in lung cancer shows promise in improving the efficacy of ...
Morningstar4d
FDA Grants Orphan Drug & Rare Pediatric Disease Designations to Infinant Health for NEC
"Securing this Orphan Drug Designation sends a positive message to the neonatology community," said Mike Johnson, CEO of Infinant Health. "The Rare Pediatric Disease Designation is an important ...
IASO Bio Receives Orphan Drug Designation from the Saudi Food and Drug Authority for Equecabtagene Autoleucel
IASO Biotherapeutics ("IASO Bio"), a biopharmaceutical company focused on the discovery, development, manufacturing, and commercialization of innovative cell therapies and biologics, today announced ...
Morningstar4d
Notice of Orphan Drug Designation for JR-446 for Mucopolysaccharidosis Type IIIB by the U.S. FDA
MEDIPAL HOLDINGS CORPORATION(TSE 7459, MEDIPAL) and JCR Pharmaceuticals Co., Ltd.(TSE 4552, JCR) today announced that the U.S. Food and Drug Administration (FDA) granted orphan drug designation ...
Neurology Advisor3d
Nitric Oxide-Regulating Therapy Gets Orphan Drug Status for Syndromic Autism
The FDA has granted Orphan Drug designation to BA-102 for the treatment of Phelan-McDermid syndrome, a rare neurodevelopmental disorder.
Notice of Orphan Drug Designation for JR-446 for Mucopolysaccharidosis Type IIIB by the U.S. FDA
MEDIPAL and JCR announced that the U.S. FDA granted orphan drug designation (ODD) to JR-446, an investigational drug for the ...