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News 2026-07-07 20:46 UTC

Vanda Pharmaceuticals announces Rare Pediatric Disease Designation for VCA-894A

The company states: "Vanda Pharmaceuticals announced that the FDA has granted Rare Pediatric Disease Designation to VCA-894A, Vanda's investigational antisense oligonucleotide therapy for the treatment of Charcot-Marie-Tooth disease, axonal, type 2S (CMT2S), a rare, serious, and progressive inherited neurological disor