Pharmaceutical company
Appears in 2 stories
Leads development and commercialization of ulefnersen
FUS-ALS patients have no approved drug targeting the cause of their disease. On September 22, an experimental therapy cleared a key hurdle: a Phase 3 trial showed ulefnersen, an RNA-targeted drug, improved function and survival versus placebo.
Updated 2 hours ago
Markets Voyxact, the first FDA-approved APRIL inhibitor for IgAN
For decades, doctors had no drugs specifically approved for IgA nephropathy, a kidney disease that pushes up to half of patients toward kidney failure within ten years of diagnosis. Since December 2021, the FDA has approved five.
Updated Jun 1
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