Gene Therapy: What Does It Mean For Rare Disease Communities

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

Rare diseases and the Bespoke Gene Therapy Consortium: Max Presa, Ph.D.

The Jackson Laboratory

Boy with ultra-rare disease begins groundbreaking gene therapy

CBC News: The National

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

Broad Institute

The Promises and Pitfalls of Gene Therapy for Rare Diseases

Gene Therapy: Your Questions Answered

National Organization for Rare Disorders (NORD)

Increase in use of gene therapy for rare diseases

Associated Press

Gene Therapy and Platform Approaches to Treating Rare Diseases

Fibrodysplasia Ossificans Progressiva (FOP): The Human Story of a Rare Genetic Condition

Untold Chronicles

Scientists meet on using gene therapy to cure rare diseases

Al Jazeera English

Understanding Biology to Investigate Potential Treatments for Rare Diseases

The Challenge to Develop and Commercialize Gene-Based Therapies for Rare Diseases

Development of Gene Therapy for Rare Diseases

CMT Research Foundation

RettEd Webinar: Gene Therapy in Rare Disease

International Rett Syndrome Foundation

Ottawa baby pioneers gene therapy for rare disease

Life After Gene Therapy

National Organization for Rare Disorders (NORD)

Gene Therapy Treatment Update - Platforms of Hope

Global Genes - Allies in Rare Disease

Understanding the Gene Therapy Process and Aftercare

National Organization for Rare Disorders (NORD)

Gene therapy into brain helps kid with rare disease

Associated Press

Curing Rare Disease Is Possible | Lynn Hopkins | TEDxBelmontShore