Rare Disease Resource Library

Advocates, Medical Professionals, Patient & Caregiver, Researchers, Gene Therapy, Collection, Education, Research,

October 17, 2022

ASGCT Gene & Cell Therapy Glossary

Advocates, Medical Professionals, Patient & Caregiver, Researchers, Gene Therapy, Collection, Education, Research,

ASGCT Gene & Cell Therapy Education: Information for patients and the public about gene therapy, including a clinical trials finder database

Medical Professionals, Patient & Caregiver, Gene Therapy, Collection, Research,

Gene Therapy 101 Workshop (2022)

Advocates, Patient Organizations, Researchers, Advocacy, Regulatory, Research & Drug Development, Collection, Publication, Education, Policy,

Orphan Drug Trends in the United States 2018

Medical Professionals, Researchers, RDCA-DAP, Research & Drug Development, Collection, Research,

Accelerating Rare Disease Drug Development: RDCA-DAP – Shortening the Timeline for Developing New Treatments for Rare Diseases

Medical Professionals, Researchers, RDCA-DAP, Research & Drug Development, Collection, Research,

Accelerating Rare Disease Drug Development: Rare Disease Challenges in Each Step of the Drug Development Process.

Medical Professionals, Researchers, RDCA-DAP, Research & Drug Development, Collection, Research,

Accelerating Rare Disease Drug Development: The Drug Development Process

Advocates, Patient & Caregiver, Patient Organizations, Students & Volunteers, Rare Disease Day, Collection, Other,

October 14, 2022

Rare Disease Day Zoom Backgrounds

Merchandise, Rare Disease Day, Collection,

Rare Disease Day Store

Advocates, Patient Organizations, Researchers, Legislative Advocacy, Research & Drug Development, Collection, Research,

September 28, 2022

Study on Orphan Drug Costs and Use