Comprehensive long-term efficacy and safety of recombinant human alpha-mannosidase (velmanase alfa) treatment in patients with alpha-mannosidosis.
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ABSTRACT:
SUBMITTER: Lund AM
PROVIDER: S-EPMC6326957 | biostudies-literature | 2018 Nov
REPOSITORIES: biostudies-literature
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Comprehensive long-term efficacy and safety of recombinant human alpha-mannosidase (velmanase alfa) treatment in patients with alpha-mannosidosis.
Lund Allan M AM Borgwardt Line L Cattaneo Federica F Ardigò Diego D Geraci Silvia S Gil-Campos Mercedes M De Meirleir Linda L Laroche Cécile C Dolhem Philippe P Cole Duncan D Tylki-Szymanska Anna A Lopez-Rodriguez Monica M Guillén-Navarro Encarna E Dali Christine I CI Héron Bénédicte B Fogh Jens J Muschol Nicole N Phillips Dawn D Van den Hout J M Hannerieke JMH Jones Simon A SA Amraoui Yasmina Y Harmatz Paul P Guffon Nathalie N
Journal of inherited metabolic disease 20180503 6
<h4>Introduction</h4>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).<h4>Methods</h4>Patient data (n = 33; 14 adults, 19 paediatric) from the clinical development programme for velmanase alfa were integrated in this prospectively-designed analysis of long-term efficacy and safety. Patients who participated in the phase I/II or phase III trials and we ...[more]
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