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- BioMarin's BMN-401 fails key endpoint in ENPP1 deficiency Phase 3 trialAn experimental therapy, BMN 401, was tested in a Phase 3 study for ENPP1 deficiency, a rare disease. The drug missed one of its two co-primary endpoints, a result that regulators consider critical…
- Relay Therapeutics' zovegalisib shows 60% response in mid‑stage trial for rare vascular anomaliesRelay Therapeutics reported early data from a phase 2 trial of its experimental drug zovegalisib in patients with vascular anomalies, a group of rare disorders characterized by abnormal blood vesse…