Leukodystrophy, Metachromatic
An autosomal recessive metabolic disease caused by a deficiency of CEREBROSIDE-SULFATASE leading to intralysosomal accumulation of cerebroside sulfate (SULFOGLYCOSPHINGOLIPIDS) in the nervous system and other organs. Pathological features include diffuse demyelination, and metachromatically-staining granules in many cell types such as the GLIAL CELLS. There are several allelic and nonallelic forms with a variety of neurological symptoms.
Drugs by status
Approved for this indication (1)
Phase 3 trials (4)
Phase 2 trials (15)
Phase 1 trials (2)
Other trials (4)
Organizations
Organization Involved with Phase 3 Indications (4)
Organization Involved with Phase 2 Indications (15)
- Shire
- Zymenex
- Fondazione Telethon
- Duke University
- Guangzhou Medical University
- Institut National de la Santé Et de la Recherche Médicale, France
- University of Louisville
- University of Copenhagen
- The University of Hong Kong
- Saint Vincent de Paul Hospital, Paris
- PhaseOne Trials
- University of Paris
- Shenzhen University
- European Leukodystrophy Association
- China Medical University
Organization Involved with Phase 1 Indications (2)
Organization Involved with Other Experimental Indications (4)