Please provide your email address to receive an email when new articles are posted on . A nonprofit focused on treatments and cures for Charcot-Marie-Tooth disease will partner with a biotechnology ...

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 ...

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), ...