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

Abstract Background 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 effic...

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Bibliographic Details
Main Authors: Barry J. Byrne, Tarekegn Geberhiwot, Bruce A. Barshop, Richard Barohn, Derralynn Hughes, Drago Bratkovic, Claude Desnuelle, Pascal Laforet, Eugen Mengel, Mark Roberts, Peter Haroldsen, Kristin Reilley, Kala Jayaram, Ke Yang, Liron Walsh, on behalf of the POM-001/002 Investigators
Format: Article
Language:English
Published: BMC 2017-08-01
Series:Orphanet Journal of Rare Diseases
Subjects:
Reveglucosidase alfa
Late-onset Pompe disease
Enzyme replacement therapy
Pharmacokinetics
Safety
Efficacy
Online Access:http://link.springer.com/article/10.1186/s13023-017-0693-2

Internet

http://link.springer.com/article/10.1186/s13023-017-0693-2

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