Ultra-Rare Disease

Boy with ultra-rare disease begins groundbreaking gene therapy

CBC News: The National

Toddler Is 1 of Only People in World With Ultra-Rare Disease

Family races against time to raise $3m to treat son’s ultra-rare disease

Al Jazeera English

The future of new treatments for ultra-rare diseases | Rob Collin | TEDxVenlo

Family of toddler with ultra-rare disease sees signs treatment may work

Emma's match: How connecting patients with ultra-rare disorders can uncover the roots of disease

Broad Institute

Toddler with ultra-rare disease released from Boston hospital

WCVB Channel 5 Boston

Heartwarming: Cancer patients share a special bond

Hendricks County toddler diagnosed with "ultra-rare" disease, family advocating for research

Better FDA Guidance for Ultra-Rare Diseases Needed

One child's ultra rare disease involving "dark spots on his brain"

The Dallas Morning News

Rare and Ultra Rare Diseases | GCT Breaks Through

World Medical Innovation Forum

Exciting Research Into The Ultra-Rare Epilepsy: Lafora Disease - Jordi Duran

Epilepsy Sparks Insights

Oregon family battles ultra-rare disease

What if we could end the rare disease diagnostic odyssey?

"No cure:" Toronto toddler battles ultra-rare genetic disorder

South Florida family shares story of son with ultra-rare genetic disorder

Ultra Rare IBD's

National Bleeding Disorders Foundation

Bringing new Hope to Families Living with Rare Disease

University Hospitals

Rare Disease Resources