TY  - JOUR
AU  - Borgwardt, Line
AU  - Guffon, Nathalie
AU  - Amraoui, Yasmina
AU  - Dali, Christine I
AU  - De Meirleir, Linda
AU  - Gil-Campos, Mercedes
AU  - Heron, Bénédicte
AU  - Geraci, Silvia
AU  - Ardigo, Diego
AU  - Cattaneo, Federica
AU  - Fogh, Jens
AU  - Van den Hout, J M Hannerieke
AU  - Beck, Michael
AU  - Jones, Simon A
AU  - Tylki-Szymanska, Anna
AU  - Haugsted, Ulla
AU  - Lund, Allan M
PY  - 2018
DO  - 10.1007/s10545-018-0185-0
UR  - http://hdl.handle.net/10668/12519
T2  - Journal of inherited metabolic disease
AB  - 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. Twenty-five patients were randomised to weekly 1 mg/kg...
LA  - en
PB  - Wiley
KW  - Adolescent
KW  - Adult
KW  - Child
KW  - Child, preschool
KW  - Double-blind method
KW  - Enzyme replacement therapy
KW  - Europe
KW  - Female
KW  - Humans
KW  - Male
KW  - Quality of life
KW  - Recombinant proteins
KW  - Severity of illness index
KW  - Treatment outcome
KW  - Young adult
KW  - alpha-Mannosidase
KW  - alpha-Mannosidosis
TI  - 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.
TY  - research article
VL  - 41
ER  -