Vanda Pharmaceuticals Inc.'s antisense oligonucleotide (ASO) VCA-894A has been awarded orphan drug designation by the FDA for the treatment of Charcot-Marie-Tooth disease, axonal, type 2S (CMT2S), ...
EN001, a novel therapy under development by ENCell, has been granted Orphan Drug Designation by the U.S. FDA for the treatment of Charcot-Marie-Tooth (CMT) disease. Phase 1 clinical trial confirmed ...
BRISBANE, Calif. , Oct. 13, 2025 /PRNewswire/ -- ReviR Therapeutics, a biotech company focused on developing novel treatments for neurogenetic diseases, announced today that the U.S. Food and Drug ...
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