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

ConclusionsThese findings support the utility of VA for the treatment of AM, with more evident benefit over time and when treatment is started in the paediatric age.
Source: Journal of Inherited Metabolic Disease - Category: Internal Medicine Source Type: research