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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’s Biologics Branch Scores Wins Against Unproven Stem Cell Clinics, Says ExpertProfessor Paul Knoepfler, a cell‑biology professor at UC Davis, writes that the FDA’s biologics division has recently taken concrete steps to curb the proliferation of unproven stem‑cell clinics in…