TY  - JOUR
AU  - Lund, Allan M
AU  - Borgwardt, Line
AU  - Cattaneo, Federica
AU  - Ardigo, Diego
AU  - Geraci, Silvia
AU  - Gil-Campos, Mercedes
AU  - De Meirleir, Linda
AU  - Laroche, Cecile
AU  - Dolhem, Philippe
AU  - Cole, Duncan
AU  - Tylki-Szymanska, Anna
AU  - Lopez-Rodriguez, Monica
AU  - Guillen-Navarro, Encarna
AU  - Dali, Christine I
AU  - Heron, Benedicte
AU  - Fogh, Jens
AU  - Muschol, Nicole
AU  - Phillips, Dawn
AU  - Van den Hout, J M Hannerieke
AU  - Jones, Simon A
AU  - Amraoui, Yasmina
AU  - Harmatz, Paul
AU  - Guffon, Nathalie
PY  - 2018
DO  - 10.1007/s10545-018-0175-2
UR  - http://hdl.handle.net/10668/12421
T2  - Journal of inherited metabolic disease
AB  - 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). Patient data (n = 33; 14 adults, 19 paediatric) from...
LA  - en
PB  - John Wiley & Sons
KW  - Alpha-mannosidosis
KW  - Enzyme replacement therapy
KW  - Integrated analysis
KW  - Lysosomal storage disorder
KW  - Recombinant human alpha-mannosidase
KW  - Velmanase alfa
KW  - Activities of daily living
KW  - Adolescent
KW  - Adult
KW  - Child
KW  - Enzyme replacement therapy
KW  - Europe
KW  - Female
KW  - Follow-up studies
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  - Comprehensive long-term efficacy and safety of recombinant human alpha-mannosidase (velmanase alfa) treatment in patients with alpha-mannosidosis.
TY  - research article
VL  - 41
ER  -