Rare Disease Clinical Trials
Connect with groundbreaking research for over 7,000 rare diseases. Access gene therapies, orphan drugs, and innovative treatments offering new hope.
Breakthrough Therapies Now Recruiting
CRISPR Gene Editing
Sickle cell, beta-thalassemia, Leber congenital amaurosis
AAV Gene Therapy
Duchenne MD, SMA, hemophilia
RNA Therapeutics
Huntington's, ALS, rare epilepsies
Browse Trials by Disease Category
Hematologic Conditions
- • Sickle Cell Disease
- • Beta Thalassemia
- • Hemophilia A & B
- • Von Willebrand Disease
Rare Neurological Diseases
- • ALS (Lou Gehrig's)
- • Huntington's Disease
- • Spinal Muscular Atrophy
- • Friedreich's Ataxia
Neuromuscular Disorders
- • Duchenne MD
- • Becker MD
- • Myasthenia Gravis
- • Pompe Disease
Primary Immunodeficiencies
- • SCID
- • CGD
- • X-linked Agammaglobulinemia
- • DiGeorge Syndrome
Rare Pediatric Diseases
- • Batten Disease
- • Progeria
- • Rett Syndrome
- • Dravet Syndrome
Revolutionary Treatment Approaches
Comprehensive Patient Support
Centers of Excellence
Connect with specialized rare disease centers and expert physicians nationwide
Find Centers →Financial Assistance
Travel grants, lodging support, and treatment cost coverage for trial participants
Get Support →Patient Advocacy
Connect with patient organizations and support groups for your condition
Join Groups →Expanded Access Programs
Can't join a clinical trial? Expanded access (compassionate use) programs may provide investigational treatments outside of trials for serious rare diseases.
- ✓For life-threatening conditions without approved treatments
- ✓Available when clinical trial enrollment isn't possible
- ✓Requires physician and FDA approval
Quick Eligibility Check
Stories of Hope
"Gene therapy gave my daughter her life back"
After years of decline from SMA, a single gene therapy infusion changed everything. She's now walking, running, and living like any other 8-year-old.
- Maria T., SMA Parent
"CRISPR trial cured my sickle cell disease"
No more pain crises, no more hospitalizations. The gene editing trial I joined two years ago has completely transformed my quality of life.
- James K., Sickle Cell Warrior
Join the Rare Disease Registry
Register once and get matched with multiple trials. We'll notify you when new studies open for your condition. Over 50,000 patients already registered.
Join RegistryYour Rare Disease Doesn't Define Your Future
With 95% of rare diseases lacking FDA-approved treatments, clinical trials offer the best hope for breakthrough therapies. Find your trial today.