Niemann-Pick Diseases
A group of autosomal recessive disorders in which harmful quantities of lipids accumulate in the viscera and the central nervous system. They can be caused by deficiencies of enzyme activities (SPHINGOMYELIN PHOSPHODIESTERASE) or defects in intracellular transport, resulting in the accumulation of SPHINGOMYELINS and CHOLESTEROL. There are various subtypes based on their clinical and genetic differences.
Subtype terms (3)
More specific conditions that are subtypes of Niemann-Pick Diseases in the MeSH hierarchy.
- Niemann-Pick Disease, Type A
1 drug approved
- Niemann-Pick Disease, Type B
5 drugs (4 approved, 1 experimental)
- Niemann-Pick Disease, Type C
12 drugs (8 approved, 4 experimental)
Drugs by status
Approved for this indication (2)
Phase 3 trials (4)
Phase 2 trials (1)
Phase 1 trials (3)
Organizations
Organization Involved with Phase 4 Indications (1)
Organization Involved with Phase 3 Indications (6)
Organization Involved with Phase 2 Indications (13)
- Cyclotherapeutics
- National Institutes of Health (NIH)
- National Human Genome Research Institute (NHGRI)
- Cornell University
- Duke University
- Johns Hopkins University
- University of Louisville
- Columbia University
- Jiao Tong University
- Salford Royal Foundation NHS Trust
- University of Oxford
- Washington University in St. Louis
- IntraBio Inc
Organization Involved with Phase 1 Indications (4)