Verified charities working on Rare disease research

7 organisations tagged Rare disease research. Every one links to a filing you can open yourself — we don't rank them.

• ALS Therapy Development Institute — First non-profit biotech 100% focused on ALS drug discovery — pre-clinical research.
• Children's Tumor Foundation — Funds neurofibromatosis (NF) research — venture-philanthropy model accelerating drug development.
• Crohn's & Colitis Foundation — Largest US IBD-focused charity — research, patient education, advocacy.
• Cystic Fibrosis Foundation — Funded the venture-philanthropy R&D pipeline that produced Trikafta (CF therapy).
• Lupus Foundation of America — Funds lupus research, advocates for patients, runs national support network.
• Sickle Cell Disease Association of America — National federation supporting people with sickle cell disease — research, advocacy, education.
• Fondazione Telethon — Italian rare-disease research foundation — pioneered gene therapy with Strimvelis (ADA-SCID).