Impact of imiglucerase supply constraint on the therapeutic management and course of disease in French patients with Gaucher disease type 1

BACKGROUND: In 2009, a worldwide supply constraint of imiglucerase led to treatment modifications or interruptions for patients with Gaucher disease (GD) type 1. In France, joint treatment recommendations were issued to protect the most vulnerable patients. This observational study evaluated the imp...

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Detalhes bibliográficos
Publicado no:Orphanet J Rare Dis
Main Authors: Stirnemann, Jérôme, Rose, Christian, Serratrice, Christine, Dalbies, Florence, Lidove, Olivier, Masseau, Agathe, Pers, Yves-Marie, Baron, Camille, Belmatoug, Nadia
Formato: Artigo
Idioma:Inglês
Publicado em: BioMed Central 2015
Assuntos:
Research
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC4434532/
https://ncbi.nlm.nih.gov/pubmed/25968608
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s13023-015-0275-0
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