Breakthrough · March 27, 2026
Nonprofit Uses AI to Match Old Drugs with Rare Diseases in New Treatment Push
A novel drug repurposing strategy offers new hope for treating rare diseases by identifying existing medications with untapped the...
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....
Technology · February 23, 2026
FDA Streamlines Approval Process to Accelerate Gene Therapy Access
FDA streamlines approval process for gene therapies, accelerating life-saving treatments to patients....
Treatment · February 23, 2026
FDA Unveils Plan To Speed Approval Of Rare Disease Treatments
FDA announces plans to accelerate approval timelines for rare disease treatments, benefiting patients in need....