A novel gene therapy has demonstrated impressive early results in a small sample of 3 children with Duchenne muscular dystrophy.
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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.
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Latest Updated Articles
After Long Wait, Several Novel Therapies Are on the Horizon for ALSPublished: January 4th 2018 | Updated:
BHV-0223 Expanded Access Program Opened for ALS, as FDA Application Is PreparedPublished: May 9th 2018 | Updated:
Gene Therapy Shows Robust Activity for Duchenne Muscular DystrophyPublished: June 23rd 2018 | Updated:
FDA Grants Priority Review to Fremanezumab for Migraine PreventionPublished: January 3rd 2018 | Updated:
FDA Green Lights Amantadine Extended-Release Tablets for Parkinson DiseasePublished: February 19th 2018 | Updated:
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