Mucopolysaccharidoses
Group of lysosomal storage diseases each caused by an inherited deficiency of an enzyme involved in the degradation of glycosaminoglycans (mucopolysaccharides). The diseases are progressive and often display a wide spectrum of clinical severity within one enzyme deficiency.
Subtype terms (6)
More specific conditions that are subtypes of Mucopolysaccharidoses in the MeSH hierarchy.
- Mucopolysaccharidosis I
27 drugs (17 approved, 10 experimental)
- Mucopolysaccharidosis II
26 drugs (13 approved, 13 experimental)
- Mucopolysaccharidosis III
23 drugs (8 approved, 15 experimental)
- Mucopolysaccharidosis IV
4 drugs (3 approved, 1 experimental)
- Mucopolysaccharidosis VI
18 drugs (12 approved, 6 experimental)
- Mucopolysaccharidosis VII
11 drugs (8 approved, 3 experimental)
Drugs by status
Approved for this indication (3)
Phase 4 trials (5)
Phase 3 trials (9)
Phase 2 trials (17)
- acetylcysteine
- vosoritide
- hgt-1410
- allogeneic stem cells
- heparan sulfate
- allogeneic hematopoietic stem cells
- sbc-103
- celecoxib
- cyclosporine
- alemtuzumab
- saf-301
- scaav9.u1a.hsgsh
- losartan
- autologous plasmablasts
- frozen autologous cd34+ hematopoietic stem and progenitor cells genetically modified with the lentiviral vector idua lv, encoding for the α-l-iduronidase cdna
- sobi-003
- sig-005
Other trials (1)
Organizations
Organization Involved with Phase 4 Indications (9)
Organization Involved with Phase 3 Indications (60)
- Swedish Orphan Biovitrum
- Green Cross Corporation
- Aldagen
- JCR Pharmaceuticals Co., Ltd.
- LYSOGENE
- Sponsor GmbH
- University of Pennsylvania
- Baylor University
- University of London
- Harvard University
- Duke University
- Federal University of Mato Grosso do Sul
- Florida International University
- 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, Irvine
- University of California, San Francisco
- +30 more
Organization Involved with Phase 2 Indications (19)
- Abeona Therapeutics
- Alexion
- ArmaGen, Inc
- Inventiva Pharma
- Regenxbio Inc.
- Fondazione Telethon
- National Institutes of Health (NIH)
- University of Pittsburgh
- Centro Hospitalar do Porto
- Federal University of Health Science of Porto Alegre
- Ohio State University
- University of Santiago de Compostela
- University of Adelaide
- University of Lyon
- Vita-Salute University of Milano. Italy
- Orchard Therapeutics Limited
- CTI Clinical Trial and Consulting Services
- The Isaac Foundation
- Denali Therapeutics Inc.
Organization Involved with Phase 1 Indications (7)
Organization Involved with Other Experimental Indications (5)