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Evaluation of disease burden and response to treatment in adults with type 1 gaucher disease using a validated disease severity scoring system (DS3)

BACKGROUND: GD1-DS3 is an integrated assessment of type 1 Gaucher disease (GD1) burden based on bone, hematologic and visceral domains. We investigated this disease severity scoring system (DS3) methodology for initial assessment, long-term follow-up and evaluation of treatment responses. METHODS: W...

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Detalles Bibliográficos
Autores principales:	Weinreb, Neal J., Finegold, David N., Feingold, Eleanor, Zeng, Zhen, Rosenbloom, Barry E., Shankar, Suma P., Amato, Dominick
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	BioMed Central 2015
Materias:	Research
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4471923/ https://www.ncbi.nlm.nih.gov/pubmed/25994334 http://dx.doi.org/10.1186/s13023-015-0280-3

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