Vanda Pharmaceuticals Gets FDA Rare Pediatric Disease Designation for CMT2S Therapy
() -- Vanda Pharmaceuticals (VNDA) said Tuesday the Food and Drug Administration (FDA) granted Rare Pediatric Disease Designation to VCA-894A, its investigational antisense oligonucleotide therapy for Charcot-Marie-Tooth disease, axonal, type 2S (CMT2S), a rare inherited neurological disorder that progressively causes