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Long Term Follow-Up of 103 Untreated Adult Patients with Type 1 Gaucher Disease

The introduction of disease-specific therapy for patients with type I Gaucher disease (GD1) was a revolution in the management of patients, but not without cost. Thus, the management of mildly affected patients is still debated. We herein report a long-term follow-up (median (range) of 20 (5–58) yea...

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Detalles Bibliográficos
Autores principales: Dinur, Tama, Zimran, Ari, Becker-Cohen, Michal, Arkadir, David, Cozma, Claudia, Hovakimyan, Marina, Oppermann, Sebastian, Demuth, Laura, Rolfs, Arndt, Revel-Vilk, Shoshana
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2019
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6832634/
https://www.ncbi.nlm.nih.gov/pubmed/31614613
http://dx.doi.org/10.3390/jcm8101662