Safety, tolerability, pharmacokinetics, pharmacodynamics, and exploratory efficacy of the novel enzyme replacement therapy avalglucosidase alfa (neoGAA) in treatment-naïve and alglucosidase alfa-treated patients with late-onset Pompe disease: A phase 1, open-label, multicenter, multinational, ascending dose study-Reference-Cited by-同舟云学术

Safety, tolerability, pharmacokinetics, pharmacodynamics, and exploratory efficacy of the novel enzyme replacement therapy avalglucosidase alfa (neoGAA) in treatment-naïve and alglucosidase alfa-treated patients with late-onset Pompe disease: A phase 1, open-label, multicenter, multinational, ascending dose study

Published:2019-03 Issue:3 Volume:29 Page:167-186
ISSN:0960-8966
Container-title:Neuromuscular Disorders
language:en
Short-container-title:Neuromuscular Disorders

Author:

Pena Loren D.M.^ORCID,Barohn Richard J.,Byrne Barry J.,Desnuelle Claude^ORCID,Goker-Alpan Ozlem,Ladha Shafeeq,Laforêt Pascal,Mengel Karl Eugen,Pestronk Alan,Pouget Jean,Schoser Benedikt,Straub Volker^ORCID,Trivedi Jaya,Van Damme Philip^ORCID,Vissing John,Young Peter,Kacena Katherine,Shafi Raheel,Thurberg Beth L.,Culm-Merdek Kerry,van der Ploeg Ans T.

Publisher

Elsevier BV

Subject

Genetics (clinical),Neurology (clinical),Neurology,Pediatrics, Perinatology and Child Health

Reference60 articles.

1. Glycogen storage disease type II: acid alpha-glucosidase (acid maltase) deficiency;Hirschhorn,2001

2. Pompe's disease;van der Ploeg;Lancet,2008

3. Enzyme replacement therapy for Pompe disease;Angelini;Curr Neurol Neurosci Rep,2012

4. Pathology of skeletal muscle cells in adult-onset glycogenosis type II (Pompe disease): ultrastructural study;Lewandowska;Folia Neuropathol,2008

5. Timing of diagnosis of patients with Pompe disease: data from the Pompe registry;Kishnani;Am J Med Genet A,2013

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