Related references
Note: Only part of the references are listed.Facial-muscle weakness, speech disorders and dysphagia are common in patients with classic infantile Pompe disease treated with enzyme therapy
C. M. van Gelder et al.
JOURNAL OF INHERITED METABOLIC DISEASE (2012)
36 months observational clinical study of 38 adult Pompe disease patients under alglucosidase alfa enzyme replacement therapy
Caroline Regnery et al.
JOURNAL OF INHERITED METABOLIC DISEASE (2012)
Observational clinical study in juvenile-adult glycogenosis type 2 patients undergoing enzyme replacement therapy for up to 4 years
C. Angelini et al.
JOURNAL OF NEUROLOGY (2012)
Effect of enzyme therapy and prognostic factors in 69 adults with Pompe disease: an open-label single-center study
Juna M. de Vries et al.
ORPHANET JOURNAL OF RARE DISEASES (2012)
Recombinant human acid alpha-glucosidase (rhGAA) in adult patients with severe respiratory failure due to Pompe disease
David Orlikowski et al.
NEUROMUSCULAR DISORDERS (2011)
Survival and associated factors in 268 adults with Pompe disease prior to treatment with enzyme replacement therapy
Deniz Guengoer et al.
ORPHANET JOURNAL OF RARE DISEASES (2011)
Long-term observational, non-randomized study of enzyme replacement therapy in late-onset glycogenosis type II
Bruno Bembi et al.
JOURNAL OF INHERITED METABOLIC DISEASE (2010)
Enzyme replacement therapy with alglucosidase alfa in 44 patients with late-onset glycogen storage disease type 2: 12-month results of an observational clinical trial
S. Strothotte et al.
JOURNAL OF NEUROLOGY (2010)
A Randomized Study of Alglucosidase Alfa in Late-Onset Pompe's Disease.
Ans T. van der Ploeg et al.
NEW ENGLAND JOURNAL OF MEDICINE (2010)
Early Treatment With Alglucosidase Alfa Prolongs Long-Term Survival of Infants With Pompe Disease
Priya S. Kishnani et al.
PEDIATRIC RESEARCH (2009)
Lysosomal storage disease 2 - Pompe's disease
Ans T. van der Ploeg et al.
LANCET (2008)
Immortal time bias in observational studies of drug effects
Samy Suissa
PHARMACOEPIDEMIOLOGY AND DRUG SAFETY (2007)
Chinese hamster ovary cell-derived recombinant human acid α-glucosidase in infantile-onset Pompe disease
Priya Sunil Kishnani et al.
JOURNAL OF PEDIATRICS (2006)
Disease severity in children and adults with Pompe disease related to age and disease duration
MLC Hagemans et al.
NEUROLOGY (2005)
Clinical manifestation and natural course of late-onset Pompe's disease in 54 Dutch patients
MLC Hagemans et al.
BRAIN (2005)
Late-onset Pompe disease primarily affects quality of life in physical health domains
MLC Hagemans et al.
NEUROLOGY (2004)
Enzyme therapy for Pompe disease with recombinant human α-glucosidase from rabbit milk
JMP Van den Hout et al.
JOURNAL OF INHERITED METABOLIC DISEASE (2001)
Recombinant human α-glucosidase from rabbit milk in Pompe patients
H Van den Hout et al.
LANCET (2000)
Randomized, controlled trials, observational studies, and the hierarchy of research designs.
J Concato et al.
NEW ENGLAND JOURNAL OF MEDICINE (2000)