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News about "Rare Pediatric Disease Designation "
Galibra Neuroscience has secured both Orphan Drug Designation and Rare Pediatric Disease Designation from the U.S. FDA for its investigational gene therapy for SSADH deficiency, strengthening regulatory support for a potential disease-modifying treatment aimed at addressing the genetic cause of the rare neurological disorder.
Rare Pediatric Disease Designation | 06/08/2026 | By News Bureau
Atossa Therapeutics Receives FDA Orphan Drug Designation for Duchenne Therapy
Atossa Therapeutics has received US Food and Drug Administration (FDA) Orphan Drug Designation (ODD) for (Z)-Endoxifen for the treatment of Duchenne Muscular Dystrophy, further supporting the company’s development programme in rare paediatric neuromuscular diseases.
Rare Pediatric Disease Designation | 17/01/2026 | By News Bureau | 165
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