Business Wire

ENCell’s EN001 Receives Orphan Drug Designation from the U.S. FDA for Charcot-Marie-Tooth Disease

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 ...
KXAN

ReviR Therapeutics Receives Orphan Drug Designation from FDA for RTX-117 to Treat Charcot-Marie-Tooth Disease

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 ...
ksn.com

NMD Pharma Announces FDA Orphan Drug Designation Granted to NMD670 for the Treatment of Patients with Charcot-Marie-Tooth Disease

This is the 2 nd orphan drug designation granted to the skeletal muscle-specific drug therapy NMD670 Under this newly granted orphan drug designation, NMD670 is currently being evaluated in a Phase 2 ...