Comprehensive long-term efficacy and safety of recombinant human alpha-mannosidase (velmanase alfa) treatment in patients with alpha-mannosidosis-Reference-Cited by-同舟云学术

Comprehensive long-term efficacy and safety of recombinant human alpha-mannosidase (velmanase alfa) treatment in patients with alpha-mannosidosis

Published:2018-05-03 Issue:6 Volume:41 Page:1225-1233
ISSN:0141-8955
Container-title:Journal of Inherited Metabolic Disease
language:en
Short-container-title:J Inherit Metab Dis

Author:

Lund Allan M.¹²,Borgwardt Line¹²³,Cattaneo Federica⁴,Ardigò Diego⁴,Geraci Silvia⁴,Gil-Campos Mercedes⁵,De Meirleir Linda⁶,Laroche Cécile⁷,Dolhem Philippe⁸,Cole Duncan⁹,Tylki-Szymanska Anna¹⁰,Lopez-Rodriguez Monica¹¹,Guillén-Navarro Encarna¹²,Dali Christine I.¹,Héron Bénédicte¹³,Fogh Jens¹⁴,Muschol Nicole¹⁵,Phillips Dawn¹⁶,Van den Hout J. M. Hannerieke¹⁷,Jones Simon A.¹⁸,Amraoui Yasmina¹⁹,Harmatz Paul²⁰,Guffon Nathalie²¹

Affiliation:

1. Departments of Paediatrics and Adolescent Medicine; Centre for Inherited Metabolic Diseases; Copenhagen Denmark

2. ; Department of Clinical Genetics, Centre for Inherited Metabolic Diseases, Copenhagen University Hospital; Rigshospitalet; Copenhagen Denmark

3. ; Center for Genomic Medicine, Copenhagen University Hospital; Rigshospitalet; Copenhagen Denmark

4. ; Chiesi Farmaceutici S.p.A; Parma Italy

5. ; Unidad de Metabolismo e Investigación Pediátrica, Hospital Universitario Reina Sofía, IMIBIC; Universidad de Córdoba, CIBERObn; Córdoba Spain

6. ; Paediatric Neurology and Metabolism; Universitair Ziekenhuis; Brussels Belgium

7. Limoges Hospital; Limoges France

8. ; Centre Hospitalier de Saint-Quentin; Saint-Quentin France

9. ; Department of Medical Biochemistry and Immunology; University Hospital of Wales; Cardiff Wales UK

10. ; Department of Paediatric, Nutrition and Metabolic Diseases; The Children's Memorial Health Institute; Warsaw Poland

11. ; Hospital Central Cruz Roja; Madrid Spain

12. Medical Genetics Section, Hospital Clínico Universitario Virgen de la Arrixaca, IMIB-Arrixaca, CIBERER-ISCIII; Madrid Spain

13. ; Service de Neuropédiatrie, Centre de Référence des Maladies Lysosomales, and Sorbonne Université, GRC n°19, pathologies Congénitales du Cervelet-LeucoDystrophies, AP-HP, Hôpital Armand Trousseau; Paris F-75012 France

14. ; Zymenex A/S; Hillerød Denmark

15. ; International Center for Lysosomal Disorders; University Medical Center Hamburg-Eppendorf; Hamburg Germany

16. ; Evidera; Bethesda MD USA

17. ; Center for Lysosomal and Metabolic Diseases (department of Pediatrics); Erasmus MC University Medical Center - Sophia Children's Hospital; Rotterdam The Netherlands

18. ; Manchester Centre for Genomic Medicine; Central Manchester University Hospitals NHS Foundation Trust; Manchester UK

19. ; Center for Pediatric and Adolescent Medicine, Villa Metabolica; University Medical Center Mainz; Mainz Germany

20. ; UCSF Benioff Children's Hospital Oakland; Oakland CA USA

21. ; Centre de Référence des Maladies Héréditaires du Métabolisme; Hôpital Femme Mère Enfant; Lyon France

Funder

FP7 International Cooperation

Zymenex A/S

Chiesi Farmaceutici

Publisher

Wiley

Subject

Genetics(clinical),Genetics

Link

http://link.springer.com/article/10.1007/s10545-018-0175-2/fulltext.html

Reference20 articles.

1. Enzyme replacement therapy for alpha-mannosidosis: 12 months follow-up of a single Centre, randomised, multiple dose study;Borgwardt;J Inherit Metab Dis,2013

2. Alpha-mannosidosis - a review of genetic, clinical findings and options of treatment;Borgwardt;Pediatr Endocrinol Rev,2014

3. Enzyme replacement therapy in children and adolescents with α-mannosidosis: an 18-month follow-up;Borgwardt;J Inborn Errors Metab Screen 2: Abstract #1180,2014b

4. Alpha-mannosidosis: correlation between phenotype, genotype and mutant MAN2B1 subcellular localisation;Borgwardt;Orphanet J Rare Dis,2015

5. Borgwardt L Guffon N Amraoui Y 2018 Efficacy and safety of velmanase alfa in the treatment of patients with alpha-mannosidosis: results from the core and extension phase analysis of a phase III multicentre, double-blind, randomised, placebo-controlled trial 10.1007/s10545-018-0185-0.

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