Advocates, Medical Professionals, Pediatric, Physician, Researchers, Advocacy, Research & Drug Development, Publication, Medical Affairs, Research, December 13, 2024 How To Advocate For Rare Disease-Related Policies: A Guide for Professionals Involved in Diagnosis, Care, or Research of Rare Diseases
Advocates, Medical Professionals, Researchers, Students & Volunteers, Rare Disease Advisory Councils (RDAC), Webinar, Policy, February 7, 2023 RDAC Stakeholders Meeting – 2022
Advocates, Medical Professionals, Patient & Caregiver, Patient Organizations, Researchers, Gene Therapy, Collection, Educational Video, November 3, 2022 Genome Editing 4 – The approval process
Advocates, Medical Professionals, Patient & Caregiver, Patient Organizations, Researchers, Gene Therapy, Collection, Educational Video, Genome Editing 3 – How do I participate and what is the role of patient organizations?
Advocates, Medical Professionals, Patient & Caregiver, Patient Organizations, Researchers, Gene Therapy, Collection, Educational Video, Genome Editing 2 – What are the safety, ethical and cost concerns
Medical Professionals, Patient & Caregiver, Researchers, Gene Therapy, Collection, Educational Video, Genome Editing 1 – What is genome editing?
Advocates, Medical Professionals, Patient & Caregiver, Researchers, RDCA-DAP, Research & Drug Development, Educational Video, Webinar, Research, October 27, 2022 What You Need to Know About the Rare Disease Cures Accelerator-Data and Analytics Platform
Medical Professionals, Patient & Caregiver, Researchers, Gene Therapy, Collection, Podcast Episode, Other, October 20, 2022 “Ungeeking The Speak: Dr Rachel Bailey Talks “Gene Therapy 101” (2021)
Medical Professionals, Researchers, Research & Drug Development, Understanding Rare Disease, PDF, Publication, Education, 2015 Neurological Rare Disease Special Report
Advocates, Patient & Caregiver, Patient Organizations, Researchers, Students & Volunteers, Advocacy, RDCA-DAP, Research & Drug Development, Infographic, PDF, Education, Policy, The Five Steps in Drug Development (Spanish version)
