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- Ultragenyx's Angelman syndrome drug fails Phase 3 trial, halting developmentUltragenyx reported that its experimental antisense oligonucleotide designed for Angelman syndrome did not meet primary endpoints in a Phase 3 study. The trial, which enrolled patients with the ra…
- FDA advisory panels for Capricor and Replimune highlight need for rare-disease specialist inputRecent FDA advisory committee meetings reviewing therapies from Capricor Therapeutics and Replimune produced markedly different outcomes, underscoring how the agency’s reliance on specialist input…