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- Ultragenyx gene therapy for Angelman syndrome fails Phase 3 trial, company to cut expensesAn experimental gene therapy targeting Angelman syndrome did not meet its primary endpoint in a Phase 3 study, according to Ultragenyx. The trial showed no difference between the therapy, GTX-102,…
- FDA requests public input to accelerate botanical drug product developmentThe U.S. Food and Drug Administration issued a request for information (RFI) to collect public comments on ways to move botanical drug products forward. Botanical drug products are derived from pl…