Breakthrough · April 18, 2026
Decades of Research Yield Transformative Cystic Fibrosis Drugs Saving Lives
An unsolved medical mystery led researchers to a life-saving breakthrough that will help patients....
Treatment · March 17, 2026
Regulators Pledge to Streamline Gene Therapy Approvals for Rare Diseases
Federal regulators commit to improving and streamlining approval processes for gene therapies treating rare diseases....
Pediatrics · March 17, 2026
Parents Push For Faster Drug Access For Children With Rare Diseases
Parents advocate for expedited access to life-saving medications for children with rare genetic diseases....