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

L Borgwardt; N Guffon; Y Amraoui; CI Dali; L de Meirleir; M Gil-Campos; B Heron; S Geraci; D Ardigò; F Cattaneo; J Fogh; Hannerieke van den Hout; M Beck; SA Jones; A Tylki-Szymanska; U Haugsted; AM Lund

doi:10.1007/s10545-018-0185-0

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

L Borgwardt, N Guffon, Y Amraoui, CI Dali, L de Meirleir, M Gil-Campos, B Heron, S Geraci, D Ardigò, F Cattaneo, J Fogh, Hannerieke van den Hout, M Beck, SA Jones, A Tylki-Szymanska, U Haugsted, AM Lund

Pediatrics

Research output: Contribution to journal › Article › Academic › peer-review

48 Citations (Scopus)

23 Downloads (Pure)

Original language	Undefined/Unknown
Pages (from-to)	1215-1223
Number of pages	9
Journal	Journal of Inherited Metabolic Disease
Volume	41
Issue number	6
DOIs	https://doi.org/10.1007/s10545-018-0185-0
Publication status	Published - 2018

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EMC OR-01

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10.1007/s10545-018-0185-0

Repub_128341_O-A.pdfFinal published version, 508 KB

http://hdl.handle.net/1765/128341

Cite this

Borgwardt, L., Guffon, N., Amraoui, Y., Dali, CI., de Meirleir, L., Gil-Campos, M., Heron, B., Geraci, S., Ardigò, D., Cattaneo, F., Fogh, J., van den Hout, H., Beck, M., Jones, SA., Tylki-Szymanska, A., Haugsted, U., & Lund, AM. (2018). 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. Journal of Inherited Metabolic Disease, 41(6), 1215-1223. https://doi.org/10.1007/s10545-018-0185-0

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author = "L Borgwardt and N Guffon and Y Amraoui and CI Dali and \{de Meirleir\}, L and M Gil-Campos and B Heron and S Geraci and D Ardig{\`o} and F Cattaneo and J Fogh and \{van den Hout\}, Hannerieke and M Beck and SA Jones and A Tylki-Szymanska and U Haugsted and AM Lund",

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Borgwardt, L, Guffon, N, Amraoui, Y, Dali, CI, de Meirleir, L, Gil-Campos, M, Heron, B, Geraci, S, Ardigò, D, Cattaneo, F, Fogh, J, van den Hout, H, Beck, M, Jones, SA, Tylki-Szymanska, A, Haugsted, U & Lund, AM 2018, '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', Journal of Inherited Metabolic Disease, vol. 41, no. 6, pp. 1215-1223. https://doi.org/10.1007/s10545-018-0185-0

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. / Borgwardt, L; Guffon, N; Amraoui, Y et al.
In: Journal of Inherited Metabolic Disease, Vol. 41, No. 6, 2018, p. 1215-1223.

Research output: Contribution to journal › Article › Academic › peer-review

TY - JOUR

T1 - 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

AU - Borgwardt, L

AU - Guffon, N

AU - Amraoui, Y

AU - Dali, CI

AU - de Meirleir, L

AU - Gil-Campos, M

AU - Heron, B

AU - Geraci, S

AU - Ardigò, D

AU - Cattaneo, F

AU - Fogh, J

AU - van den Hout, Hannerieke

AU - Beck, M

AU - Jones, SA

AU - Tylki-Szymanska, A

AU - Haugsted, U

AU - Lund, AM

PY - 2018

Y1 - 2018

U2 - 10.1007/s10545-018-0185-0

DO - 10.1007/s10545-018-0185-0

M3 - Article

C2 - 29846843

SN - 0141-8955

VL - 41

SP - 1215

EP - 1223

JO - Journal of Inherited Metabolic Disease

JF - Journal of Inherited Metabolic Disease

IS - 6

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Borgwardt L, Guffon N, Amraoui Y, Dali CI, de Meirleir L, Gil-Campos M et al. 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. Journal of Inherited Metabolic Disease. 2018;41(6):1215-1223. doi: 10.1007/s10545-018-0185-0