Ontology highlight
ABSTRACT:
SUBMITTER: Borgwardt L
PROVIDER: S-EPMC6326984 | biostudies-literature | 2018 Nov
REPOSITORIES: biostudies-literature
Borgwardt Line L Guffon Nathalie N Amraoui Yasmina Y Dali Christine I CI De Meirleir Linda L Gil-Campos Mercedes M Heron Bénédicte B Geraci Silvia S Ardigò Diego D Cattaneo Federica F Fogh Jens J Van den Hout J M Hannerieke JMH Beck Michael M Jones Simon A SA Tylki-Szymanska Anna A Haugsted Ulla U Lund Allan M AM
Journal of inherited metabolic disease 20180530 6
<h4>Introduction</h4>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.<h4>Methods</h4>Twenty-five patients were randomised to weekly 1 mg/kg VA or placebo for 52 weeks. At study conclusion, placebo patients switched to VA; 23 patients continued receiving VA in compassionate-use/follow-on studies and were evaluated in the extension phase [last observati ...[more]