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- Ionis and Otsuka's RNA therapy Ulefnersen shows functional and survival gains in rare FUS-ALS in Phase 3 trialIonis Pharmaceuticals and Otsuka Pharmaceutical reported that their antisense oligonucleotide drug Ulefnersen improved both functional impairment scores and overall survival in a Phase 3 study of p…
- Roche reports phase 3 success for sefaxersen in IgA nephropathy, eyes accelerated approvalRoche announced that its experimental drug sefaxersen met the primary endpoint in a phase 3 trial for IgA nephropathy, showing reduced proteinuria versus placebo. The interim analysis at week 37 d…Fierce Biotech · 1mo agoPositive readoutPositive