Mitochondrial Diseases
UCB Wins First FDA Approval for Ultra-Rare Mitochondrial Disease: Kygevvi for Thymidine Kinase 2 Deficiency (TK2d)
UCB; FDA approval; Kygevvi; mitochondrial disease; Thymidine Kinase 2 deficiency; TK2d; doxecitine; doxribtimine; rare diseases; Orphan Drug; Breakthrough Therapy; Priority Review; Rare Pediatric Disease
FDA Grants Accelerated Approval to Stealth BioTherapeutics’ Forzinity, the First Treatment for Barth Syndrome
FDA approval; Stealth BioTherapeutics; Forzinity; elamipretide; Barth syndrome; mitochondrial disease; accelerated approval; rare disease; pediatric disease; clinical trial