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- Relay's PI3Kα inhibitor clears efficacy bar in Phase 2 vascular anomalies studyRelay Therapeutics’ first data readout for zovegalisib in a rare genetic disease beat Wall Street expectations, expanding the PI3Kα inhibitor’s R&D prospects beyond breast cancer. The biotech on Tu…Endpoints · 2mo ago↗ source
- BioMarin's rare disease therapy shows no clinical benefit in Phase 3 testBioMarin’s enzyme replacement therapy for a rare genetic disorder called ENPP1 deficiency delivered mixed results in a late-stage study. Patients with the condition don't produce enough of the ENPP…Endpoints · 2mo ago↗ source
- STAT+: 1 in 8 women drink during pregnancy. Experts dread the consequencesMore than 1 in 8 pregnant women report drinking. Doctors warn fetal alcohol disorders may be more common than many realize, perhaps more common than autism.