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Fabry Disease
An X-linked inherited metabolic disease caused by a deficiency of lysosomal ALPHA-GALACTOSIDASE A. It is characterized by intralysosomal accumulation of globotriaosylceramide and other GLYCOSPHINGOLIPIDS in blood vessels throughout the body leading to multi-system complications including renal, cardiac, cerebrovascular, and skin disorders.
Drugs by status
Approved for this indication (4)
Phase 4 trials (4)
Phase 3 trials (2)
Phase 2 trials (11)
Phase 1 trials (3)
Other trials (6)
Organizations
Organization Involved with Phase 4 Indications (5)
Organization Involved with Phase 3 Indications (13)
Organization Involved with Phase 2 Indications (19)
- Resverlogix Corp
- National Institutes of Health (NIH)
- Baylor University
- University of London
- Duke University
- Emory University
- University of Tennessee
- University of Toronto
- University of Utah
- University of Minnesota
- Sangamo BioSciences
- Washington University in St. Louis
- Tuscon Access Center of Arizona Kidney Disease Hypertension Center
- Christus St. Patrick Hospital
- CRL/Medinet
- Queen Elizabeth Hospital NHS Foundation Trust
- Freeline Therapeutics
- AvroBio
- 4D Molecular Therapeutics
Organization Involved with Phase 1 Indications (5)
Organization Involved with Other Experimental Indications (19)
- Chiesi
- Kaiser Permanente
- Oregon Health and Science University
- Denver Nephrologists, P.C.
- University of California, Davis
- University of California, San Diego
- University of California, San Francisco
- University of Naples Frederico II
- Covance
- University of Missouri
- University Research Foundation for Lysosomal Storage Disorders
- Infusion Associates
- St. Joseph's Regional Medical Center, Wisconsin
- Mission Health, North Carolina
- Central Coast Nephrology
- Carilion New River Valley Medical Center
- Tidewater Kidney Specialists
- University of Massachusetts
- Rouen Normandy University