Gene Therapy: What Does It Mean For Rare Disease Communities

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

Gene Therapy 101: What Is It and How Can It Help Us Treat Rare Genetic Disorders?

PTC Therapeutics

Boy with ultra-rare disease begins groundbreaking gene therapy

CBC News: The National

Increase in use of gene therapy for rare diseases

Associated Press

Gene Therapy: Your Questions Answered

National Organization for Rare Disorders (NORD)

Gene Editing Technologies: Transforming Rare Disease Care

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

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

Cancer Patients and Caregivers Share their Stories | People v. Cancer

Introduction to Rare Disease Day 2020 Part 1

Coming of Age Together: Rare Disease Advocacy and Gene Therapy | Michelle Berg

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

The Jackson Laboratory

Gene therapy into brain helps kid with rare disease

Associated Press

Scientists meet on using gene therapy to cure rare diseases

Al Jazeera English

Gene Therapy and Platform Approaches to Treating Rare Diseases

Health Equity and Rare Disorders

National Organization for Rare Disorders (NORD)

2021 Reuters Cell & Gene Therapy

MDA Engage: Gene Therapy and Neuromuscular Disease

MDA

RettEd Webinar: Gene Therapy in Rare Disease

International Rett Syndrome Foundation

Supporting Our Rare Disease Community