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

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). METHODS: Patient data (n = 33; 14 adults, 19 paediatric) from the clinical development pr...

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Vydáno v:J Inherit Metab Dis
Hlavní autoři: Lund, Allan M., Borgwardt, Line, Cattaneo, Federica, Ardigò, Diego, Geraci, Silvia, Gil-Campos, Mercedes, De Meirleir, Linda, Laroche, Cécile, Dolhem, Philippe, Cole, Duncan, Tylki-Szymanska, Anna, Lopez-Rodriguez, Monica, Guillén-Navarro, Encarna, Dali, Christine I., Héron, Bénédicte, Fogh, Jens, Muschol, Nicole, Phillips, Dawn, Van den Hout, J. M. Hannerieke, Jones, Simon A., Amraoui, Yasmina, Harmatz, Paul, Guffon, Nathalie
Médium: Artigo
Jazyk:Inglês
Vydáno: Springer Netherlands 2018
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Original Article
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC6326957/
https://ncbi.nlm.nih.gov/pubmed/29725868
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1007/s10545-018-0175-2
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