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- 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
- Amgen stands by rare disease drug Tavneos amid Japan liver toxicity reportThe FDA wants it off the market. European regulators are investigating data-integrity concerns. And now, a pharma company selling the drug in Japan is warning doctors against writing new prescripti…Endpoints · 2mo ago↗ source
- FDA's unreleased Covid vaccine deaths report is published by lawmakerAn analysis launched by former FDA leaders to look into claims of pediatric deaths related to Covid-19 vaccines was published by a Republican lawmaker, months after the agency's self-imposed deadli…