A study on the safety and efficacy of reveglucosidase alfa in patients with late-onset Pompe disease
A study on the safety and efficacy of reveglucosidase alfa in patients with late-onset Pompe disease
About this item
Full title
Author / Creator
POM-001/002 Investigators , on behalf of the POM-001/002 Investigators , Byrne, Barry J , Geberhiwot, Tarekegn , Barshop, Bruce A , Barohn, Richard , Hughes, Derralynn , Bratkovic, Drago , Desnuelle, Claude , Laforet, Pascal , Mengel, Eugen , Roberts, Mark , Haroldsen, Peter , Reilley, Kristin , Jayaram, Kala , Yang, Ke and Walsh, Liron
Publisher
England: BioMed Central Ltd
Journal title
Language
English
Formats
Publication information
Publisher
England: BioMed Central Ltd
Subjects
More information
Scope and Contents
Contents
Late-onset Pompe disease is a rare genetic neuromuscular disorder caused by lysosomal acid alpha-glucosidase (GAA) deficiency that ultimately results in mobility loss and respiratory failure. Current enzyme replacement therapy with recombinant human (rh)GAA has demonstrated efficacy in subjects with late-onset Pompe disease. However, long-term effe...
Alternative Titles
Full title
A study on the safety and efficacy of reveglucosidase alfa in patients with late-onset Pompe disease
Authors, Artists and Contributors
Author / Creator
on behalf of the POM-001/002 Investigators
Byrne, Barry J
Geberhiwot, Tarekegn
Barshop, Bruce A
Barohn, Richard
Hughes, Derralynn
Bratkovic, Drago
Desnuelle, Claude
Laforet, Pascal
Mengel, Eugen
Roberts, Mark
Haroldsen, Peter
Reilley, Kristin
Jayaram, Kala
Yang, Ke
Walsh, Liron
Identifiers
Primary Identifiers
Record Identifier
TN_cdi_doaj_primary_oai_doaj_org_article_0f42cc539c044d06b2948b37cb169a2e
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_doaj_primary_oai_doaj_org_article_0f42cc539c044d06b2948b37cb169a2e
Other Identifiers
ISSN
1750-1172
E-ISSN
1750-1172
DOI
10.1186/s13023-017-0693-2
