News Medical on MSN17h
Gene therapy breakthrough offers hope for severe Dravet syndrome cases
AAV-Navβ1 gene therapy effectively reduces seizures and prolongs life in a mouse model of SCN1B-linked Dravet syndrome, ...
Biogen buys rights to Stoke’s rare epilepsy drug
In exchange for $165 million, Biogen now has access to zorevunersen, a potential first-of-its-kind treatment for Dravet syndrome.
Solid Biosciences Reports Positive Initial Clinical Data from Next-Generation Duchenne Gene Therapy Candidate SGT-003
Day 90 biopsy data reported from first 3 participants dosed in Phase 1/2 INSPIRE DUCHENNE trial —— Average microdystrophin expression of 110% ...