Rare Disease Resources
ICD Code Roadmap Everylife Resource Guide- For all who seek to better understand how diagnosis codes are assigned, updated and revised in the United States’ health information system, this ICD Code Roadmap is for you. The Roadmap provides all the information you will need to determine whether and how to get involved in the continuous process of refining the diagnostic coding system used in the U.S.
Guide to Patient Involvement in Drug Development– From the Everylife Foundation- Developed BY the community, FOR the community. This Guide is anchored in an assessment of FDA guidance on the inclusion of patient experience in therapy development for rare diseases. Whether you are a patient advocacy organization, industry partner, payer, or other critical stakeholder, the Guide is intended to embolden engagement among stakeholders.
Once Upon a Gene Podcast– True stories of raising rare kiddos, hosted by Effie Parks
Understanding Drug Development– Global Genes RARE University course
Vision Loss Resources
NEI Information for patients and families living with rare eye disorders
My Retina Tracker Registry – Open Access Genetic Testing Program hosted by Foundation Fighting Blindness
Gene Therapy 101
The American Society of Cell and Gene Therapy (ASGCT) Youtube channel– a great educational resource to learn more about gene therapy
U.S. Food and Drug Administration (FDA)- Gene Therapy Inside Out (video)
National Organization for Rare Disorders– Gene Therapy Your Questions answered (video)