Pediatric Disorder
Fortress Biotech and Sentynl Therapeutics Fail to Secure FDA Approval for CUTX-101 in Rare Pediatric Menkes Disease
Fortress Biotech; Sentynl Therapeutics; FDA Complete Response Letter; CUTX-101; Menkes disease; rare pediatric disease; manufacturing deficiencies; drug approval setback
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