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

INTRODUCTION: This phase III, double-blind, randomised, placebo-controlled trial (and extension phase) was designed to assess the efficacy and safety of velmanase alfa (VA) in alpha-mannosidosis (AM) patients. METHODS: Twenty-five patients were randomised to weekly 1 mg/kg VA or placebo for 52 weeks...

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Vydáno v:J Inherit Metab Dis
Hlavní autoři: Borgwardt, Line, Guffon, Nathalie, Amraoui, Yasmina, Dali, Christine I., De Meirleir, Linda, Gil-Campos, Mercedes, Heron, Bénédicte, Geraci, Silvia, Ardigò, Diego, Cattaneo, Federica, Fogh, Jens, Van den Hout, J. M. Hannerieke, Beck, Michael, Jones, Simon A., Tylki-Szymanska, Anna, Haugsted, Ulla, Lund, Allan M.
Médium: Artigo
Jazyk:Inglês
Vydáno: Springer Netherlands 2018
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Original Article
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC6326984/
https://ncbi.nlm.nih.gov/pubmed/29846843
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1007/s10545-018-0185-0
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