Galibra Neuroscience Receives FDA Orphan Drug and Rare Pediatric Disease Designations for SSADH Deficiency Gene Therapy

Dual designations support development of a potential first disease-modifying therapy for an ultra-rare neurometabolic disorder BOSTON, Aug. 5, 2026 /PRNewswire/ -- Galibra Neuroscience announced a significant regulatory milestone with the receipt of both Orphan Drug Designation (ODD) and...

Summary aggregated from PR Newswire's public RSS feed. The full reporting belongs to PR Newswire — please read it on their site.