Mechanistic convergence and shared therapeutic targets in Niemann-Pick disease.

Colaco, Alexandria

Mechanistic convergence and shared therapeutic targets in Niemann-Pick disease. [electronic resource] - Journal of inherited metabolic disease 05 2020 - 574-585 p. digital

Publication Type: Journal Article; Research Support, Non-U.S. Gov't

1573-2665

10.1002/jimd.12191 doi


1-Deoxynojirimycin--analogs & derivatives
ATP Binding Cassette Transporter 1--genetics
Adult
Humans
Intracellular Signaling Peptides and Proteins--genetics
Middle Aged
Niemann-Pick C1 Protein
Niemann-Pick Disease, Type C--drug therapy
Treatment Outcome