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Mucopolysaccharidosis II
Systemic lysosomal storage disease marked by progressive physical deterioration and caused by a deficiency of L-sulfoiduronate sulfatase. This disease differs from MUCOPOLYSACCHARIDOSIS I by slower progression, lack of corneal clouding, and X-linked rather than autosomal recessive inheritance. The mild form produces near-normal intelligence and life span. The severe form usually causes death by age 15.
Drugs by status
Approved for this indication (1)
Phase 4 trials (4)
Phase 3 trials (7)
Phase 2 trials (11)
Organizations
Organization Involved with Phase 4 Indications (6)
Organization Involved with Phase 3 Indications (50)
- Green Cross Corporation
- JCR Pharmaceuticals Co., Ltd.
- University of Pennsylvania
- Baylor University
- University of London
- Harvard University
- Federal University of Mato Grosso do Sul
- Harbin Medical University
- Los Angeles Biomedical Research Institute
- RWTH Aachen University
- Justus Liebig University Giessen
- University of Hamburg
- University of Iowa
- University of Manchester
- University of Melbourne
- University of Milan
- University of Nebraska
- University of Nottingham
- University of Padova
- University of Toronto
- University of Bath
- St. Louis University
- University of California, San Francisco
- University of Cambridge
- University of Naples Frederico II
- University of Utah
- Federal University of Rio de Janeiro
- Comprehensive Cancer Centers of Nevada
- University of Minnesota
- Badalona Hospital Germans Trias i Pujol
- +20 more
Organization Involved with Phase 2 Indications (5)
Organization Involved with Phase 1 Indications (4)