Implications of the choroid plexus in Niemann-Pick disease Type C neuropathogenesis. [PDF]
van Gool R +18 more
europepmc +1 more source
Plasma phosphorylated-tau217 is increased in Niemann-Pick disease type C. [PDF]
Gonzalez-Ortiz F +7 more
europepmc +1 more source
Clinical Evaluation of N-Acetyl-l-Leucine in Niemann-Pick Disease Type C: A Critical Perspective. [PDF]
Lopez G, Holgado C.
europepmc +1 more source
Reduced native T<sub>1</sub> on cardiac magnetic resonance imaging as a novel marker of myocardial involvement in Niemann-Pick disease type B. [PDF]
Redzepi B +8 more
europepmc +1 more source
Utility of 24(S)-hydroxycholesterol as a proximal biomarker to monitor long-term intrathecal adrabetadex therapy in individuals with Niemann-Pick disease, type C1. [PDF]
Porter FD +8 more
europepmc +1 more source
Role of mTORC1 signaling in postnatal microglia activation preceding neurodegeneration in a mouse model for Niemann-Pick disease Type C. [PDF]
Murray CE +8 more
europepmc +1 more source
Prevalence of Neutralizing Antibodies to AAV2 and AAV9 in Individuals with Niemann-Pick Disease, Type C1. [PDF]
Mylvara AV +4 more
europepmc +1 more source
Inability of α-cyclodextrins to accommodate cholesterol potentially underlies their lack of efficacy and ototoxicity in Niemann-Pick disease type C treatment. [PDF]
Yamada Y +22 more
europepmc +1 more source

