Resources
Rare Diseases often go unnoticed.
Our job is to ensure the opposite is true. We are proactive in our goal to understand rare diseases and to provide patient associations with every opportunity to collaborate with us. We will ensure that we efficiently and continually share information about our work on treatment options, listen to the needs of the community, and focus our efforts on achieving the best outcomes on their behalf.
Rare Disease Umbrella Organizations
Fabry
Alpha-Mannosidosis
Leber’s Hereditary Optic Neuropathy
Nephropathic Cystinosis
Thalassemia
ADA-SCID
