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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Veröffentlicht in:Orphanet J Rare Dis
Hauptverfasser: Stirnemann, Jérôme, Rose, Christian, Serratrice, Christine, Dalbies, Florence, Lidove, Olivier, Masseau, Agathe, Pers, Yves-Marie, Baron, Camille, Belmatoug, Nadia
Format: Artigo
Sprache:Inglês
Veröffentlicht: BioMed Central 2015
Schlagworte:
Research
Online Zugang: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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