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- Silence Therapeutics’ Phase 2 Trial Shows Strong Efficacy of divesiran in Polycythemia VeraSilence Therapeutics reported that its siRNA candidate divesiran met the primary endpoint in a phase 2 study of patients with polycythemia vera. The trial enrolled 48 participants and 88% of those…
- Biogen’s ALS drug shows symptom improvement in some patients three years after approvalBiogen’s antisense therapy tofersen, marketed as Qalsody, received FDA approval three years ago as the first treatment to target a genetic form of ALS linked to the SOD1 gene. Since then, a number…