Breakthrough · March 20, 2026
CRISPR Gene Editing Saves Infant in Pioneering One-Off Treatment for Rare Disease
Top medical advances of 2025 bring renewed optimism for treating previously intractable 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....