Results 131 to 140 of about 30,962 (231)

Discovery of Autophagy Modulators that Increase I1061T NPC1 Expression and Promote Cholesterol Efflux in Niemann-Pick Type C Patient-Derived Fibroblasts. [PDF]

ACS Chem Biol
Salkovski M, Arrieche Suarez A, Gao Q, Hippman RS, Petros ZA, Korkmaz-Vaisys MA, Gerlach EM, Alvarado-Cartagena YM, Nguyen TTA, Pavlinov I, Ilnytska O, Storch J, Cologna SM, Aldrich LN.   +13 more
europepmc   +1 more source

N-acetyl-L-leucine for Niemann-Pick type C: a multinational double-blind randomized placebo-controlled crossover study. [PDF]

Trials, 2023
Fields T, M Bremova T, Billington I, Churchill GC, Evans W, Fields C, Galione A, Kay R, Mathieson T, Martakis K, Patterson M, Platt F, Factor M, Strupp M.   +13 more
europepmc   +1 more source

Exploration of Bromodomain Proteins as Drug Targets for Niemann-Pick Type C Disease. [PDF]

Int J Mol Sci
Parente M, Barthelemy A, Tonini C, Caputo S, Sacchi A, Leone S, Segatto M, Pfrieger FW, Pallottini V.   +8 more
europepmc   +1 more source

Treatment trials in Niemann-Pick type C disease. [PDF]

Metab Brain Dis, 2021
Sitarska D, Tylki-Szymańska A, Ługowska A.   +2 more
europepmc   +1 more source

Case Report: Interleukin-23 blockade achieves sustained remission in Niemann-Pick type C-associated Crohn's disease refractory to anti-tumor necrosis factor therapy. [PDF]

Front Immunol
Kakiuchi T, Shimada S, Zhang Y, Nomura M, Yoshiura M.   +4 more
europepmc   +1 more source

Niemann-Pick type C disease as proof-of-concept for intelligent biomarker panel selection in neurometabolic disorders. [PDF]

Dev Med Child Neurol, 2022
Papandreou A, Doykov I, Spiewak J, Komarov N, Habermann S, Kurian MA, Mills PB, Mills K, Gissen P, Heywood WE, Clinical cohort recruitment and characterization group.   +10 more
europepmc   +1 more source

Advances in mass spectrometry of lipids for the investigation of Niemann-pick type C disease. [PDF]

Lipids Health Dis
Javanshad R, Li W, Pathmasiri KC, Cologna SM.   +3 more
europepmc   +1 more source

Investigation of 2-Hydroxypropyl-β-Cyclodextrin Treatment in a Neuronal-Like Cell Model of Niemann-Pick Type C Using Quantitative Proteomics. [PDF]

J Am Soc Mass Spectrom, 2023
Cougnoux A, Pergande MR, Serna-Perez F, Cologna SM.   +3 more
europepmc   +1 more source

The impact of profit taxation on capitalized investment with options to delay and divest [PDF]

In entrepreneurial decisions making uncertain future profits often are a main characteristics of real investment opportunities. If investors can react to uncertainty the degree of irreversibility and timing flexibility inherent in the available project ...
Schneider, Georg, Sureth, Caren
core  

Species-specific differences in NPC1 protein trafficking govern therapeutic response in Niemann-Pick type C disease. [PDF]

JCI Insight, 2022
Schultz ML, Schache KJ, Azaria RD, Kuiper EQ, Erwood S, Ivakine EA, Farhat NY, Porter FD, Pathmasiri KC, Cologna SM, Uhler MD, Lieberman AP.   +11 more
europepmc   +1 more source