Results 121 to 130 of about 24,478,248 (209)

Central sleep apnea and daytime sleepiness in Niemann-Pick type C disease: a report of 2 cases. [PDF]

J Clin Sleep Med, 2023
Zhang Y, Cheng Y, Li N, Hou Y, Jiao L, Yuan Y, Wang L, Huang Z, Han F, Li H, Zhan S.   +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

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

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

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

Hypometabolism and atrophy patterns associated with Niemann-Pick type C. [PDF]

EJNMMI Res
Silva-Rodríguez J, Castro C, Cortés J, Arias M, Pubul V, Moscoso A, Grothe MJ, Reynes-Llompart G, Rodríguez-Bel L, Gascon-Bayarri J, Sobrido MJ, Aguiar P.   +11 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

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 FDA HAS APPROVED A DRUG AGAINST NIEMANN–PICK DISEASE, TYPE C

Педиатрическая фармакология
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Article Editorial
doaj  

Niemann-Pick Type C Disease: From Variant of Uncertain Significance to Pathogenic Variant. [PDF]

Mov Disord Clin Pract
Rezende LB, Aguiar CN, Balbino JMV, Maia DP, Maciel RH, Camargos ST, Cardoso F.   +6 more
europepmc   +1 more source