Diagnosis features of pediatric Gaucher disease patients in the era of enzymatic therapy, a national-base study from the Spanish Registry of Gaucher Disease

Andrade-Campos, Marcio; Alfonso, Pilar; Irun, Pilar; Armstrong, Judith; Calvo, Carmen; Dalmau, Jaime; Domingo, Maria-Rosario; Barbera, Jose-Luis; Cano, Horacio; Fernandez-Galán, Maria-Angeles; Franco, Rafael; Gracia, Inmaculada; Gracia-Antequera, Miguel; Ibañez, Angela; Lendinez, Francisco; Madruga, Marcos; Martin-Hernández, Elena; O’Callaghan, Maria del Mar; del Soto, Alberto Pérez; del Prado, Yolanda Ruiz; Sancho-Val, Ignacio; Sanjurjo, Pablo; Pocovi, Miguel; Giraldo, Pilar

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BioMed Central, Orphanet Journal of Rare Diseases, 1(12)

DOI: 10.1186/s13023-017-0627-z

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Diagnosis features of pediatric Gaucher disease patients in the era of enzymatic therapy, a national-base study from the Spanish Registry of Gaucher Disease

Journal article published in 2017 by Marcio Andrade-Campos, Pilar Alfonso, Pilar Irun, Judith Armstrong, Carmen Calvo, Jaime Dalmau, Maria-Rosario Domingo

, Jose-Luis Barbera, Horacio Cano, Maria-Angeles Fernandez-Galán, Rafael Franco, Inmaculada Gracia, Miguel Gracia-Antequera, Angela Ibañez, Francisco Lendinez and other authors.

This paper is made freely available by the publisher.

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Diagnosis features of pediatric Gaucher disease patients in the era of enzymatic therapy, a national-base study from the Spanish Registry of Gaucher Disease