Orphan Disease Center Resources
Here you will find our Orphan Disease Center resources. Please see below for resources on orphan drug development, specific disease resources, along with ODC produced videos of patient stories and past Million Dollar Bike Ride teasers.
Orphan Drug Development
Disease Resources
What is Krabble Disease?
What is Gene Therapy for GM1 Gangliosidosis?
Crigler-Najjar and the Potential for Gene Therapy
Maple Syrup Urine Disease (MSUD)
What is Gene Therapy for MPS I?
HoFH and the Potential for Gene Therapy
Hispanic Society of Rare Disease - Sociedad Hispana de Enfermedades Raras (SHER)
The Hispanic Society of Rare Diseases is an association that promotes public awareness on rare diseases and provides information and resources in Spanish. We seek to be a link between organizations, scientists and health professionals focused on rare diseases and Hispanic families.
Visit: https://www.hispanicsocietyrarediseases.org
See below write up and video on SHER for more information:
A helping hand in disease
To recognize World Rare Disease Day in 2023, an organization is born with the mission of bringing science to the Latino community.
#ShirleyRadio - Salud en Onda: las enfermedades raras
Orphan Disease Center Videos
Quincy’s Story
Million Dollar Bike Ride 2019
Virtual Million Dollar Bike Ride 2020
Million Dollar Bike Ride 2018