Staff Writer

A novel gene therapy has demonstrated impressive early results in a small sample of 3 children with Duchenne muscular dystrophy.

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.

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.

Two more novel therapies are quickly following the first approval in 2 decades for ALS.

The FDA has granted a priority review designation to fremanezumab for the prevention of migraines.