Diagnosis, quality of life, and treatment of patients with Hunter syndrome in the French healthcare system: a retrospective observational study

被引:0
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作者
Nathalie Guffon
Bénédicte Heron
Brigitte Chabrol
François Feillet
Vincent Montauban
Vassili Valayannopoulos
机构
[1] Centre de Référence des Maladies Héréditaires du Métabolisme,
[2] Hospices Civils de Lyon Hôpital Femme Mère Enfants,undefined
[3] Service de Neurologie Pédiatrique,undefined
[4] Centre de Référence des Maladies Lysosomales,undefined
[5] CHU Trousseau,undefined
[6] APHP,undefined
[7] Service de Neurologie Pédiatrique,undefined
[8] Centre de Référence des Maladies Héréditaires du Métabolisme. Hôpital d’Enfants,undefined
[9] CHU La Timone,undefined
[10] Centre de Référence des Maladies Héréditaires du Métabolisme,undefined
[11] Service de Médecine Infantile,undefined
[12] CHU Brabois Enfants,undefined
[13] Shire,undefined
[14] 88 rue du Dôme,undefined
[15] Centre de Référence Maladies Métaboliques de l’enfant et de l’adulte,undefined
[16] Hôpital Universitaire Necker-Enfants Malades et Institut IMAGINE,undefined
[17] Current address: Baxter Healthcare,undefined
来源
Orphanet Journal of Rare Diseases | / 10卷
关键词
Hunter syndrome; Lysosomal storage disease; Iduronate-2-sulfatase; Enzyme replacement therapy; Quality of life;
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