Resource Library

Get Involved, Legislative Advocacy, Regulatory, Understanding Rare Disease

Published February 11, 2020

Your Voice Matters: How to Engage with the FDA

Genetic Testing, Research & Drug Development, Understanding Rare Disease

Published August 31, 2018

Genetic Testing 101 for People with Rare Diseases

Gene Therapy, Research & Drug Development, Understanding Rare Disease

Published July 18, 2018

Gene Therapy: Your Questions Answered

Gene Therapy, Research & Drug Development, Understanding Rare Disease

Published January 10, 2020

Life After Gene Therapy

Gene Therapy, Research & Drug Development, Understanding Rare Disease

Published December 5, 2019

Understanding Gene Therapy Process and Aftercare

Gene Therapy, Regulatory, Research & Drug Development, Understanding Rare Disease

Published December 13, 2019

FDA’s Role in Gene Therapy

Gene Therapy, Research & Drug Development, Understanding Rare Disease

Published October 7, 2019

The Science Behind Gene Therapy

Gene Therapy, Research & Drug Development, Understanding Rare Disease

Published September 19, 2019

Gene Therapy Yesterday, Today and Tomorrow

Advocacy, Understanding Rare Disease

Published August 6, 2019

Self-Advocacy and Care Coordination

Understanding Rare Disease

Published June 26, 2019

How Specialty Pharmacies Support the Rare Community

Understanding Rare Disease

Published June 26, 2019

Generic Drugs and Biosimilars

Advocacy, Insurance, Research & Drug Development, Understanding Rare Disease

Published January 18, 2019

How to Make Your Health Insurance Work For You

Understanding Rare Disease

Published November 15, 2019

10 Thing You May Not Know About Rare Diseases

Understanding Rare Disease

Published March 28, 2019

10 Tips for Newly Diagnosed Rare Disease Patients and Caregivers

Understanding Rare Disease

Published November 8, 2019

Pediatric Movement Disorders: Guidance for Parents and Physicians