Enzyme replacement therapy in late-onset Pompe disease: a systematic literature review

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