Real-world safety and effectiveness of vosoritide: Results from an early access program in France

被引：0

作者：

Cormier-Daire, Valerie ^{[1
]}

Edouard, Thomas ^{[2
]}

Isidor, Bertrand ^{[3
]}

Cohen, Shelda ^{[4
]}

Mukherjee, Swati ^{[4
]}

Pimenta, Jeanne ^{[4
]}

Lhaneche, Leila ^{[5
]}

Rossi, Massimiliano ^{[6
,7
]}

Schaefer, Elise ^{[8
]}

Goodman, Erin ^{[9
]}

Sigaudy, Sabine ^{[10
]}

Baujat, Genevieve ^{[11
]}

机构：

[1] Paris Ctr Univ, Imagine Inst, Necker Enfants Malad Hosp,INSERM,UMR 1163, Dept Clin Genet,Ctr Reference Constitut Bone Dis, Paris, France

[2] Toulouse Univ Hosp, Reference Ctr Rare Dis Calcium & Phosphate Metab, Paediat Res Unit,INSERM,U1301, ERN BOND,OSCAR Network,Childrens Hosp,RESTORE, Toulouse, France

[3] CHU Nantes, Nantes, France

[4] BioMarin UK Ltd, London, England

[5] BioMarin France, Paris, France

[6] Hosp Civils Lyon, Serv Genet, Lyon, France

[7] UCBL1, INSERM, U1028, CNRS,UMR5292,CRNL,GENDEV Team, Lyon, France

[8] Hop Hautepierre, CHU Strasbourg, Strasbourg, France

[9] BioMarin Pharmaceut Inc, Novato, CA USA

[10] Hop Timone Enfant, Dept Genet Med, Marseille, France

[11] Enfants Malad Hop Necker Enfants Malad, Hop Necker, Paris, France

来源：

HORMONE RESEARCH IN PAEDIATRICS | 2023年 / 96卷

关键词：

D O I：

暂无

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

RFC4.5

引用

页码：90 / 90

页数：1