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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...
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/PMC6832634/ https://www.ncbi.nlm.nih.gov/pubmed/31614613 http://dx.doi.org/10.3390/jcm8101662 |