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

Allan M. Lund,Line Borgwardt,Federica Cattaneo,Diego Ardigò,Silvia Geraci,Mercedes Gil-Campos,Linda De Meirleir,Cécile Laroche,Philippe Dolhem,Duncan Cole,Anna Tylki-Szymańska,Monica Lopez-Rodriguez,Encarna Guillén-Navarro,Christine I. Dali,Bénédicte Héron,Jens Fogh,Nicole Muschol,Dawn Phillips,J. M. Hannerieke Van den Hout,Simon A. Jones,Yasmina Amraoui,Paul Harmatz,Nathalie Guffon

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

2018

Introduction Long-term outcome data provide important insights into the clinical utility of enzyme replacement therapies. Such data are presented for velmanase alfa in the treatment of alpha-mannosidosis (AM).

Keywords:

Enzyme
Biology
Diabetes mellitus
Internal medicine
Alpha-mannosidosis
Endocrinology
Recombinant Human Alpha Mannosidase
Velmanase alfa
Enzyme replacement therapy
Pharmacology
in patient
outcome data
Human genetics
Bioinformatics

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