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Deniz Gungor et al.
AMERICAN JOURNAL OF MEDICAL GENETICS PART A (2013)
Enzyme replacement therapy in late-onset Pompe disease: a systematic literature review
Antonio Toscano et al.
JOURNAL OF NEUROLOGY (2013)
Enzyme replacement therapy and fatigue in adults with Pompe disease
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MOLECULAR GENETICS AND METABOLISM (2013)
Pain in adult patients with Pompe disease A cross-sectional survey
D. Gungor et al.
MOLECULAR GENETICS AND METABOLISM (2013)
The Rasch-built Pompe-specific Activity (R-PAct) scale
N. A. M. E. van der Beek et al.
NEUROMUSCULAR DISORDERS (2013)
Impact of enzyme replacement therapy on survival in adults with Pompe disease: results from a prospective international observational study
Deniz Gungor et al.
ORPHANET JOURNAL OF RARE DISEASES (2013)
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)
Open-label extension study following the Late-Onset Treatment Study (LOTS) of alglucosidase alfa
Ans T. van der Ploeg et al.
MOLECULAR GENETICS AND METABOLISM (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)
24-Months results in two adults with Pompe disease on enzyme replacement therapy
Stefan Vielhaber et al.
CLINICAL NEUROLOGY AND NEUROSURGERY (2011)
Burden of illness of Pompe disease in patients only receiving supportive care
Tim A. Kanters et al.
JOURNAL OF INHERITED METABOLIC DISEASE (2011)
Recombinant human acid alpha-glucosidase (rhGAA) in adult patients with severe respiratory failure due to Pompe disease
David Orlikowski et al.
NEUROMUSCULAR DISORDERS (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)
Glycogen storage disease type II (Pompe disease) - influence of enzyme replacement therapy in adults
T. Merk et al.
EUROPEAN JOURNAL OF NEUROLOGY (2009)
Progress in enzyme replacement therapy in glycogen storage disease type II
Corrado Angelini et al.
Therapeutic Advances in Neurological Disorders (2009)
Clinical features of late-onset Pompe disease: A prospective cohort study
John H. J. Wokke et al.
MUSCLE & NERVE (2008)
Impact of late-onset Pompe disease on participation in daily life activities: Evaluation of the Rotterdam Handicap Scale
M. L. C. Hagemans et al.
NEUROMUSCULAR DISORDERS (2007)
Fatigue: an important feature of late-onset Pompe disease
Marloes L. C. Hagemans et al.
JOURNAL OF NEUROLOGY (2007)
Course of disability and respiratory function in untreated late-onset Pompe disease
MLC Hagemans et al.
NEUROLOGY (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)
Psychometric evaluation of a new handicap scale in immune-mediated polyneuropathies
ISJ Merkies et al.
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