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-Reference-Cited by-同舟云学术

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

Published:2018-05-30 Issue:6 Volume:41 Page:1215-1223
ISSN:0141-8955
Container-title:Journal of Inherited Metabolic Disease
language:en
Short-container-title:J Inherit Metab Dis

Author:

Borgwardt Line¹²³,Guffon Nathalie⁴,Amraoui Yasmina⁵,Dali Christine I.¹,De Meirleir Linda⁶,Gil-Campos Mercedes⁷,Heron Bénédicte⁸,Geraci Silvia⁹,Ardigò Diego⁹,Cattaneo Federica⁹,Fogh Jens¹⁰,Van den Hout J. M. Hannerieke¹¹,Beck Michael¹²,Jones Simon A.¹³,Tylki-Szymanska Anna¹⁴,Haugsted Ulla¹⁵,Lund Allan M.¹³

Affiliation:

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

2. ; Center for Genomic Medicine; Copenhagen University Hospital; Rigshospitalet Copenhagen Denmark

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

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

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

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

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

8. Department of Pediatric Neurology, Reference Center for Lysosomal Diseases; Trousseau Hospital, APHP, and GRC ConCer-LD, Sorbonne Universities, UPMC University 06; Paris France

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

10. ; Zymenex A/S; Hillerød Denmark

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

12. ; Institute of Human Genetics; University Medical Center; Mainz Germany

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

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

15. ; Department of Occupational Therapy and Physiotherapy; Copenhagen University Hospital; Rigshospitalet Copenhagen Denmark

Funder

FP7 International Cooperation

Zymenex A/S

Chiesi Farmaceutici

Publisher

Wiley

Subject

Genetics(clinical),Genetics

Link

http://link.springer.com/article/10.1007/s10545-018-0185-0/fulltext.html

Reference17 articles.

1. Natural history of alpha mannosidosis a longitudinal study;Beck;Orphanet J Rare Dis,2013

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

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

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

5. Cerebral magnetic resonance spectroscopy demonstrates long-term effect of bone marrow transplantation in α-Mannosidosis;Danielsen;JIMD Rep,2013

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3. Cost-effectiveness of velmanase alfa vs. bone marrow transplantation or no causal therapy in patients with mild to moderate alpha-mannosidosis;Biotechnology & Biotechnological Equipment;2023-10-23

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5. Can velmanase alfa be the next widespread potential therapy for alpha-mannosidosis?;International Journal of Surgery;2023-06-21