Article ID | Journal | Published Year | Pages | File Type |
---|---|---|---|---|
8342962 | Molecular Genetics and Metabolism | 2018 | 9 Pages |
Abstract
Global treatment response to velmanase alfa (defined by response to â¥2 domains comprising pharmacodynamic, functional, and quality of life outcomes) was applied post hoc to data from the pivotal placebo-controlled rhLAMAN-05 study and to the longer-term integrated data from all patients in the clinical development program (rhLAMAN-10). After 12â¯months of treatment, a global treatment response was achieved by 87% of patients receiving velmanase alfa (nâ¯=â¯15) compared with 30% of patients receiving placebo (nâ¯=â¯10). Longer-term data from all patients in the clinical program (nâ¯=â¯33) showed 88% of patients were global responders, including all (100%) pediatric patients (nâ¯=â¯19) and the majority (71%) of adult patients (nâ¯=â¯14). The responder analysis model demonstrates a clinically meaningful treatment effect with velmanase alfa and supports the early initiation and continued benefit of longer-term treatment of all patients with alpha-mannosidosis with this ERT.
Keywords
QOLMCIDDMDBOT-2FVCCLN2IgGGTRVASEQ-5D-5LQuality of life6MWT6-min walking testEnzyme-replacement therapyenzyme replacement therapystandard deviationimmunoglobulin GMinimal Clinically Important DifferenceDuchenne muscular dystrophybody mass indexBMIforced vital capacitypharmacodynamicvisual analog scaleMucopolysaccharidosisMPsERT
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Authors
Paul Harmatz, Federica Cattaneo, Diego Ardigò, Silvia Geraci, Julia B. Hennermann, Nathalie Guffon, Allan Lund, Christian J. Hendriksz, Line Borgwardt,