A Cystic Fibrosis drug targeting the basic defect that causes the condition has been shown to be safe and effective in newborns aged four weeks and above, new research involving RCSI University of Medicine and Health Sciences and Children’s Health Ireland has found.
Ultragenyx reports early success with Angelman syndrome drug, backing up 2022 acquisition
Ultragenyx has found early success with its experimental treatment for the rare disease Angelman syndrome, supporting the company’s decision to acquire its development partner two
April 15, 2024