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Rare Diseases Research
- Home
- Research News
- Faculty
-
Rare Diseases
- Alpha-1 Antitrypsin Deficiency
- Amyotrophic Lateral Sclerosis (ALS)
- Canavan Disease
- CDKL5 Disorder
- Chronic Granulomatous
- Cockayne Syndrome
- Cystic Fibrosis
- Down Syndrome
- Facioscapulohumeral Muscular Dystrophy
- Fanconi Anemia
- Fragile X Syndrome
- Friedreich’s Ataxia
- Frontotemporal Dementia
- Gaucher Disease
- GM3 Synthase Deficiency
- Hereditary Tyrosinemia Type 1
- Leber Congenital Amaurosis
- Huntington’s Disease
- Metabolic Diseases
- Retinitis Pigmentosa
- Rett Syndrome
- Severe Chronic Neutropenia
- Sandhoff Disease
- Tay-Sachs
- Blog
- Donate Now
- Pilot Grant Program