Yahoo Finance

Vanda Pharmaceuticals Announces FDA Rare Pediatric Disease Designation for Investigational Therapy for Charcot-Marie-Tooth Disease Type 2S

The above button links to Coinbase. Yahoo Finance is not a broker-dealer or investment adviser and does not offer securities or cryptocurrencies for sale or facilitate trading. Coinbase pays us for ...
Yahoo Finance

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 ...
BioWorld

Vanda's ASO VCA-894A awarded US orphan drug designation for Charcot-Marie-Tooth disease type 2S

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), ...
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 ...
Healio

FDA grants orphan drug designation to RNA therapy for Charcot-Marie-Tooth disease type 1A

Please provide your email address to receive an email when new articles are posted on . The FDA has granted orphan drug designation to DTx-1252, an investigational small interfering RNA therapeutic to ...
Morningstar%2c Inc.

Vanda Pharmaceuticals Announces FDA Rare Pediatric Disease Designation for Investigational Therapy for Charcot-Marie-Tooth Disease Type 2S

WASHINGTON, July 7, 2026 /PRNewswire/ -- Vanda Pharmaceuticals Inc. (Vanda) (Nasdaq: VNDA) today announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease ...
Morningstar%2c Inc.

ReviR Therapeutics Receives FDA Rare Pediatric Disease Designation for RTX-117 in Charcot-Marie-Tooth Disease

Designation supports the global development of RTX-117 and may provide potential future access to a transferable Priority Review Voucher, subject to approval and applicable requirements BRISBANE, ...