Enzyme replacement therapy in late-onset Pompe disease: a systematic literature review
Published 2012 View Full Article
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Title
Enzyme replacement therapy in late-onset Pompe disease: a systematic literature review
Authors
Keywords
Late-onset Pompe disease (LOPD), Glycogen storage disease type 2, Enzyme replacement therapy, Alglucosidase alfa, Systematic review
Journal
JOURNAL OF NEUROLOGY
Volume 260, Issue 4, Pages 951-959
Publisher
Springer Nature
Online
2012-08-27
DOI
10.1007/s00415-012-6636-x
References
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Related references
Note: Only part of the references are listed.- Toward deconstructing the phenotype of late-onset Pompe disease
- (2012) Angela Schüller et al. AMERICAN JOURNAL OF MEDICAL GENETICS PART C-SEMINARS IN MEDICAL GENETICS
- 36 months observational clinical study of 38 adult Pompe disease patients under alglucosidase alfa enzyme replacement therapy
- (2012) Caroline Regnery et al. JOURNAL OF INHERITED METABOLIC DISEASE
- 24-Months results in two adults with Pompe disease on enzyme replacement therapy
- (2011) Stefan Vielhaber et al. CLINICAL NEUROLOGY AND NEUROSURGERY
- Adult Pompe disease: Clinical manifestations and outcome of the first Greek patients receiving enzyme replacement therapy
- (2011) G.K. Papadimas et al. CLINICAL NEUROLOGY AND NEUROSURGERY
- The clinical and electrodiagnostic characteristics of Pompe disease with post-enzyme replacement therapy findings
- (2011) Lisa D. Hobson-Webb et al. CLINICAL NEUROPHYSIOLOGY
- Pompe Disease: From New Views on Pathophysiology to Innovative Therapeutic Strategies
- (2011) Giancarlo Parenti et al. CURRENT PHARMACEUTICAL BIOTECHNOLOGY
- Effects of enzyme replacement therapy on five patients with advanced late-onset glycogen storage disease type II: a 2-year follow-up study
- (2011) Yoshihiko Furusawa et al. JOURNAL OF INHERITED METABOLIC DISEASE
- Observational clinical study in juvenile-adult glycogenosis type 2 patients undergoing enzyme replacement therapy for up to 4 years
- (2011) C. Angelini et al. JOURNAL OF NEUROLOGY
- First experience with enzyme replacement therapy during pregnancy and lactation in Pompe disease
- (2011) Juna M. de Vries et al. MOLECULAR GENETICS AND METABOLISM
- Effect of aerobic and resistance exercise training on late-onset Pompe disease patients receiving enzyme replacement therapy
- (2011) Gerasimos Terzis et al. MOLECULAR GENETICS AND METABOLISM
- Increased inspiratory and expiratory muscle strength following respiratory muscle strength training (RMST) in two patients with late-onset Pompe disease
- (2011) Harrison N. Jones et al. MOLECULAR GENETICS AND METABOLISM
- Rapid progressive course of later-onset Pompe disease in Chinese patients
- (2011) Chih-Chao Yang et al. MOLECULAR GENETICS AND METABOLISM
- Enhanced efficacy of enzyme replacement therapy in Pompe disease through mannose-6-phosphate receptor expression in skeletal muscle
- (2011) Dwight D. Koeberl et al. MOLECULAR GENETICS AND METABOLISM
- Consensus treatment recommendations for late-onset Pompe disease
- (2011) Edward J. Cupler et al. MUSCLE & NERVE
- Recombinant human acid alpha-glucosidase (rhGAA) in adult patients with severe respiratory failure due to Pompe disease
- (2011) David Orlikowski et al. NEUROMUSCULAR DISORDERS
- Changes in nutritional status and body composition during enzyme replacement therapy in adult-onset type II glycogenosis
- (2010) S. Ravaglia et al. EUROPEAN JOURNAL OF NEUROLOGY
- Changes in skeletal muscle qualities during enzyme replacement therapy in late-onset type II glycogenosis: temporal and spatial pattern of mass vs. strength response
- (2010) Sabrina Ravaglia et al. JOURNAL OF INHERITED METABOLIC DISEASE
- Long-term observational, non-randomized study of enzyme replacement therapy in late-onset glycogenosis type II
- (2010) Bruno Bembi et al. JOURNAL OF INHERITED METABOLIC DISEASE
- Gel-mediated Delivery of AAV1 Vectors Corrects Ventilatory Function in Pompe Mice With Established Disease
- (2010) Cathryn S Mah et al. MOLECULAR THERAPY
- Effect of enzyme therapy in juvenile patients with Pompe disease: A three-year open-label study
- (2010) C.I. van Capelle et al. NEUROMUSCULAR DISORDERS
- A Randomized Study of Alglucosidase Alfa in Late-Onset Pompe's Disease
- (2010) Ans T. van der Ploeg et al. NEW ENGLAND JOURNAL OF MEDICINE
- 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
- (2009) S. Strothotte et al. JOURNAL OF NEUROLOGY
- The Pharmacological Chaperone N-butyldeoxynojirimycin Enhances Enzyme Replacement Therapy in Pompe Disease Fibroblasts
- (2009) Caterina Porto et al. MOLECULAR THERAPY
- Glycoengineered Acid α-Glucosidase With Improved Efficacy at Correcting the Metabolic Aberrations and Motor Function Deficits in a Mouse Model of Pompe Disease
- (2009) Yunxiang Zhu et al. MOLECULAR THERAPY
- A novel mutation of the GAA gene in a Finnish late-onset pompe disease patient: Clinical phenotype and follow-up with enzyme replacement therapy
- (2009) Mari P. Korpela et al. MUSCLE & NERVE
- Progress in enzyme replacement therapy in glycogen storage disease type II
- (2009) Corrado Angelini et al. Therapeutic Advances in Neurological Disorders
- Glycogen storage disease type II (Pompe disease) - influence of enzyme replacement therapy in adults
- (2008) T. Merk et al. EUROPEAN JOURNAL OF NEUROLOGY
- Modulation of glycogen synthesis by RNA interference: towards a new therapeutic approach for glycogenosis type II
- (2008) Gaelle Douillard-Guilloux et al. HUMAN MOLECULAR GENETICS
- Eight years experience with enzyme replacement therapy in two children and one adult with Pompe disease
- (2008) C.I. van Capelle et al. NEUROMUSCULAR DISORDERS
- Rate of disease progression during long-term follow-up of patients with late-onset Pompe disease
- (2008) N.A.M.E. Van der Beek et al. NEUROMUSCULAR DISORDERS
- Early Detection of Pompe Disease by Newborn Screening Is Feasible: Results From the Taiwan Screening Program
- (2008) Y.-H. Chien et al. PEDIATRICS
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