Therapy Crazy Diseases

Boy with ultra-rare disease begins groundbreaking gene therapy

CBC News: The National

Medical Pioneer: First Patient Receives Unique Rare Disease Therapy Developed at UC San Diego

UC San Diego Health

Gene therapy into brain helps kid with rare disease

Associated Press

Focus on Rare Disease: Prime time – gene therapy for rare disorders

Broad Institute

Gene Therapy: What Does It Mean for Rare Disease Communities?

Increase in use of gene therapy for rare diseases

Associated Press

Challenges in Rare Disease Treatment

Alliance for Patient Access

19-year-old not covered for costly treatment for rare disease

CBC News: The National

TD Cowen's Ritu Bara talks Sarepta stock skyrocketing on FDA gene therapy approval

CNBC Television

Scientists meet on using gene therapy to cure rare diseases

Al Jazeera English

The Promises and Pitfalls of Gene Therapy for Rare Diseases

Four-year-old with rare disease, finally gets historic gene therapy treatment

Is shock therapy making a comeback?

Fabry disease | Center for Rare Disease Therapy at UPMC Children's Hospital of Pittsburgh

childrenshospgh

A Leap Forward in Rare Disease Treatment

ECD Global Alliance

Customized Treatment For Rare Skin Disease Through Genetics

Confronting Barriers to Rare Disease Treatment

National Press Foundation

Webinar: Rare Disease Spotlight | aHUS diagnosis, treatment, management | AKF

AGTC - Genetic Therapy For Rare Diseases

HPR

This Young Man Is One Of Two People In The World Coping With A Rare, Nameless Disease | NBC News