Effect of velmanase alfa (human recombinant alpha-mannosidase) enzyme-replacement therapy on quality of life and disease burden of patients with alpha-mannosidosis: Results from caregiver feedback

Allan Lund; Nathalie Guffon; Mercedes Gil-Campos; Frederica Cattaneo; Benedicte Heron; Anna Tylki-Szymanska; Line Borgwardt; Luc Regal; Duncan Cole; Julia Hennermann

doi:10.1016/j.ymgme.2020.12.153

Effect of velmanase alfa (human recombinant alpha-mannosidase) enzyme-replacement therapy on quality of life and disease burden of patients with alpha-mannosidosis: Results from caregiver feedback

Allan Lund, Nathalie Guffon, Mercedes Gil-Campos, Frederica Cattaneo, Benedicte Heron, Anna Tylki-Szymanska, Line Borgwardt, Luc Regal, Duncan Cole, Julia Hennermann

Pediatrics

Research output: Contribution to journal › Meeting abstract (Journal)

Original language	English
Pages (from-to)	S67-S67
Journal	Molecular Genetics and Metabolism
Volume	132
Issue number	2
DOIs	https://doi.org/10.1016/j.ymgme.2020.12.153
Publication status	Published - Feb 2021
Event	17th Annual Research Meeting of the We're-Organizing-Research-for-Lysosomal-Diseases-Symposium (WORLDS Symposium) (Online) - Duration: 7 Feb 2021 → 12 Feb 2021

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10.1016/j.ymgme.2020.12.153

Cite this

Lund, A., Guffon, N., Gil-Campos, M., Cattaneo, F., Heron, B., Tylki-Szymanska, A., Borgwardt, L., Regal, L., Cole, D., & Hennermann, J. (2021). Effect of velmanase alfa (human recombinant alpha-mannosidase) enzyme-replacement therapy on quality of life and disease burden of patients with alpha-mannosidosis: Results from caregiver feedback. Molecular Genetics and Metabolism, 132(2), S67-S67. https://doi.org/10.1016/j.ymgme.2020.12.153

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title = "Effect of velmanase alfa (human recombinant alpha-mannosidase) enzyme-replacement therapy on quality of life and disease burden of patients with alpha-mannosidosis: Results from caregiver feedback",

author = "Allan Lund and Nathalie Guffon and Mercedes Gil-Campos and Frederica Cattaneo and Benedicte Heron and Anna Tylki-Szymanska and Line Borgwardt and Luc Regal and Duncan Cole and Julia Hennermann",

year = "2021",

month = feb,

doi = "10.1016/j.ymgme.2020.12.153",

language = "English",

volume = "132",

pages = "S67--S67",

journal = "Molecular Genetics and Metabolism",

issn = "1096-7192",

publisher = "Academic Press Inc.",

number = "2",

note = "17th Annual Research Meeting of the We're-Organizing-Research-for-Lysosomal-Diseases-Symposium (WORLDS Symposium) (Online) ; Conference date: 07-02-2021 Through 12-02-2021",

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Lund, A, Guffon, N, Gil-Campos, M, Cattaneo, F, Heron, B, Tylki-Szymanska, A, Borgwardt, L, Regal, L, Cole, D & Hennermann, J 2021, 'Effect of velmanase alfa (human recombinant alpha-mannosidase) enzyme-replacement therapy on quality of life and disease burden of patients with alpha-mannosidosis: Results from caregiver feedback', Molecular Genetics and Metabolism, vol. 132, no. 2, pp. S67-S67. https://doi.org/10.1016/j.ymgme.2020.12.153

Effect of velmanase alfa (human recombinant alpha-mannosidase) enzyme-replacement therapy on quality of life and disease burden of patients with alpha-mannosidosis: Results from caregiver feedback. / Lund, Allan; Guffon, Nathalie; Gil-Campos, Mercedes et al.
In: Molecular Genetics and Metabolism, Vol. 132, No. 2, 02.2021, p. S67-S67.

Research output: Contribution to journal › Meeting abstract (Journal)

TY - JOUR

T1 - Effect of velmanase alfa (human recombinant alpha-mannosidase) enzyme-replacement therapy on quality of life and disease burden of patients with alpha-mannosidosis: Results from caregiver feedback

AU - Lund, Allan

AU - Guffon, Nathalie

AU - Gil-Campos, Mercedes

AU - Cattaneo, Frederica

AU - Heron, Benedicte

AU - Tylki-Szymanska, Anna

AU - Borgwardt, Line

AU - Regal, Luc

AU - Cole, Duncan

AU - Hennermann, Julia

PY - 2021/2

Y1 - 2021/2

U2 - 10.1016/j.ymgme.2020.12.153

DO - 10.1016/j.ymgme.2020.12.153

M3 - Meeting abstract (Journal)

SN - 1096-7192

VL - 132

SP - S67-S67

JO - Molecular Genetics and Metabolism

JF - Molecular Genetics and Metabolism

IS - 2

T2 - 17th Annual Research Meeting of the We're-Organizing-Research-for-Lysosomal-Diseases-Symposium (WORLDS Symposium) (Online)

Y2 - 7 February 2021 through 12 February 2021

ER -