← All indications
Mucopolysaccharidosis III
Mucopolysaccharidosis characterized by HEPARAN SULFATE in the urine, progressive mental retardation, mild dwarfism, and other skeletal disorders. There are four clinically indistinguishable but biochemically distinct forms, each due to a deficiency of a different enzyme.
Drugs by status
Approved for this indication (1)
Phase 3 trials (4)
Phase 2 trials (17)
Phase 1 trials (1)
Organizations
Organization Involved with Phase 3 Indications (4)
Organization Involved with Phase 2 Indications (21)
- Abeona Therapeutics
- Biomarin
- Shire
- Alexion
- Institut Pasteur
- University of Pittsburgh
- University of London
- Duke University
- Ohio State University
- University of Louisville
- University of Manchester
- University of Santiago de Compostela
- University of Adelaide
- University of California, San Francisco
- University of Minnesota
- Orchard Therapeutics Limited
- CTI Clinical Trial and Consulting Services
- Venn Life Sciences
- UniQure N.V.
- Hopitaux Universitaires Paris-Sud
- Children´s Hospital and Research Center, Oakland