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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Detalles Bibliográficos
Autores principales: Stirnemann, Jérôme, Rose, Christian, Serratrice, Christine, Dalbies, Florence, Lidove, Olivier, Masseau, Agathe, Pers, Yves-Marie, Baron, Camille, Belmatoug, Nadia
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2015
Materias:
Research
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4434532/
https://www.ncbi.nlm.nih.gov/pubmed/25968608
http://dx.doi.org/10.1186/s13023-015-0275-0

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4434532/
https://www.ncbi.nlm.nih.gov/pubmed/25968608
http://dx.doi.org/10.1186/s13023-015-0275-0

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