Browsing Tag
Angelman syndrome
3 posts
Ionis Pharmaceuticals (IONS) drops 21% as Wainua Phase 3 ATTR-CM trial fails, Alnylam Amvuttra and BridgeBio Attruby emerge as clear beneficiaries
Ionis (IONS) drops 21% as Wainua Phase 3 ATTR-CM trial fails primary endpoint. Peak sales cut to $4B. Alnylam and BridgeBio benefit from the reset.
Neuren Pharmaceuticals secures third FDA Fast Track for NNZ-2591 as shares rise 4.21%
Find out how Neuren Pharmaceuticals is expanding FDA Fast Track coverage for NNZ‑2591 across rare disorders like PMS, AS, and Pitt Hopkins.
KIK-AS clinical trial : Angelman syndrome drug candidate GTX-102 to be evaluated by GeneTx Biotherapeutics
GeneTx Biotherapeutics is set to start the KIK-AS clinical trial for evaluating GTX-102 for the treatment of Angelman…