Impact of Intravenous Trehalose Administration in Patients with Niemann–Pick Disease Types A and B

Background and Aims: Niemann–Pick disease (NPD) types A (NPA) and B (NPB) are caused by deficiency of the acid sphingomyelinase enzyme, which is encoded by the SMPD1 gene, resulting in progressive pathogenic accumulation of lipids in tissues. Trehalose has been suggested as an autophagy inducer with...

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Detalles Bibliográficos
Autores principales: Mobini, Moein, Radbakhsh, Shabnam, Kubaski, Francyne, Eshraghi, Peyman, Vakili, Saba, Vakili, Rahim, Khalili, Manijeh, Varesvazirian, Majid, Jamialahmadi, Tannaz, Alamdaran, Seyed Ali, Sayedi, Seyed Javad, Rajabi, Omid, Emami, Seyed Ahmad, Reiner, Željko, Sebkar, Amirhossein
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2022
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8745869/
https://www.ncbi.nlm.nih.gov/pubmed/35011993
http://dx.doi.org/10.3390/jcm11010247

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