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Current Challenges in Understanding the Cellular and Molecular Mechanisms in Niemann–Pick Disease Type C1

Rare diseases are a heterogeneous group of very different clinical syndromes. Their most common causes are defects in the hereditary material, and they can therefore be passed on to descendants. Rare diseases become manifest in almost all organs and often have a systemic expressivity, i.e., they aff...

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Detalles Bibliográficos
Autores principales: Bräuer, Anja U., Kuhla, Angela, Holzmann, Carsten, Wree, Andreas, Witt, Martin
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2019
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6771135/
https://www.ncbi.nlm.nih.gov/pubmed/31500175
http://dx.doi.org/10.3390/ijms20184392