Synonyms ACID SPHINGOMYELINASE DEFICIENCY, NEUROVISCERAL TYPE, ASMD, NEUROVISCERAL TYPE, A型Niemann-Pick病 + [68] |
Introduction The classic infantile form of Niemann-Pick Disease, caused by mutation in SPHINGOMYELIN PHOSPHODIESTERASE. It is characterized by accumulation of SPHINGOMYELINS in the cells of the MONONUCLEAR PHAGOCYTE SYSTEM and other cell throughout the body leading to cell death. Clinical signs include JAUNDICE, hepatosplenomegaly, and severe brain damage. |
Target |
Mechanism SMPD1 inhibitors |
Originator Org. |
Active Indication |
Inactive Indication- |
Drug Highest PhaseApproved |
First Approval Ctry. / Loc. JP |
First Approval Date28 Mar 2022 |
Target |
Mechanism PPARα agonists [+1] |
Active Org. Polaryx Therapeutics, Inc.Startup |
Originator Org. Polaryx Therapeutics, Inc.Startup |
Active Indication |
Inactive Indication |
Drug Highest PhasePhase 1 |
First Approval Ctry. / Loc.- |
First Approval Date20 Jan 1800 |
Target- |
Mechanism- |
Active Org. Polaryx Therapeutics, Inc.Startup |
Originator Org. Polaryx Therapeutics, Inc.Startup |
Active Indication |
Inactive Indication- |
Drug Highest PhasePreclinical |
First Approval Ctry. / Loc.- |
First Approval Date20 Jan 1800 |
Start Date27 Sep 2019 |
Sponsor / Collaborator |
Start Date15 Oct 2018 |
Sponsor / Collaborator |
Start Date18 Dec 2015 |
Sponsor / Collaborator |