Gene Therapy Shows Robust Activity for Duchenne Muscular Dystrophy
A novel gene therapy has demonstrated impressive early results in a small sample of 3 children with Duchenne muscular dystrophy.
BHV-0223 Expanded Access Program Opened for ALS, as FDA Application Is Prepared
An expanded access program has been opened to allow patients with ALS access to BHV-0223, a sublingual formulation of riluzole, all while Biohaven Pharmaceutical prepares a new drug application for the drug.
FDA Green Lights Amantadine Extended-Release Tablets for Parkinson Disease
The once-daily therapy will be available in 3 doses—129 mg, 193 mg, and 258 mg—giving patients flexibility in regimen setting among the options.
After Long Wait, Several Novel Therapies Are on the Horizon for ALS
Two more novel therapies are quickly following the first approval in 2 decades for ALS.
FDA Grants Priority Review to Fremanezumab for Migraine Prevention
The FDA has granted a priority review designation to fremanezumab for the prevention of migraines.