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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...
Autores principales: | , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
MDPI
2019
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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 |