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- Ultragenyx's Angelman syndrome drug fails in Phase 3 trial, showing no benefitUltragenyx announced that its experimental therapy GTX-102 for Angelman syndrome did not demonstrate any benefit compared with a sham control in a large Phase 3 trial. The drug had previously show…
- UniQure submits FDA and UK applications for first Huntington’s disease gene therapyUniQure has filed a biologics license application (BLA) with the U.S. FDA and a separate submission with the UK Medicines and Healthcare products Regulatory Agency (MHRA) for its experimental gene…