The patient journey of patients with Fabry disease, Gaucher disease and Mucopolysaccharidosis type II: A German-wide telephone survey

BACKGROUND: Lysosomal Storage Diseases (LSD) are rare and multisytemic diseases which are caused by lysosomal enzyme deficiencies leading into accumulation of waste products due to an interruption in the decomposition process. Due to the low prevalence and therefore limited disease awareness as well...

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Detalles Bibliográficos
Autores principales: Mengel, Eugen, Gaedeke, Jens, Gothe, Holger, Krupka, Simon, Lachmann, Anja, Reinke, Jörg, Ohlmeier, Christoph
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Public Library of Science 2020
Materias:
Research Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7775043/
https://www.ncbi.nlm.nih.gov/pubmed/33382737
http://dx.doi.org/10.1371/journal.pone.0244279

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