| P50 | author | Volker Straub | Q59878147 |
| | Beth L. Thurberg | Q111421204 |
| | Benedikt Schoser | Q39368899 |
| P2093 | author name string | Peter Young | |
| | John T Kissel | |
| | Alan Pestronk | |
| | Tahseen Mozaffar | |
| | Zachary Simmons | |
| | Mazen M Dimachkie | |
| | Richard J Barohn | |
| | Ozlem Goker-Alpan | |
| | Robert-Yves Carlier | |
| | Pierre G Carlier | |
| | Michela Guglieri | |
| | Stephan Wens | |
| | Stephan Wenninger | |
| | Loren D M Pena | |
| | Ans van der Ploeg | |
| | Carl Bjartmar | |
| | Matthias Boentert | |
| | Pierre-Yves Baudin | |
| | Raheel Shafi | |
| P2860 | cites work | Clinical outcomes after long-term treatment with alglucosidase alfa in infants and children with advanced Pompe disease. | Q51768443 |
| | Changes in skeletal muscle qualities during enzyme replacement therapy in late-onset type II glycogenosis: temporal and spatial pattern of mass vs. strength response. | Q51771787 |
| | Late-onset Pompe disease primarily affects quality of life in physical health domains. | Q51935783 |
| | The spectrum and diagnosis of acid maltase deficiency. | Q54261710 |
| | Trunk muscle involvement in late-onset Pompe disease: Study of thirty patients | Q58380539 |
| | Cardiac evaluation in children and adults with Pompe disease sharing the common c.-32-13T>G genotype rarely reveals abnormalities | Q64974345 |
| | Juvenile and adult-onset acid maltase deficiency in France: genotype-phenotype correlation | Q73172404 |
| | Morphological changes in muscle tissue of patients with infantile Pompe's disease receiving enzyme replacement therapy | Q73437316 |
| | Course of disability and respiratory function in untreated late-onset Pompe disease | Q82676439 |
| | Whole-body muscle MRI in 20 patients suffering from late onset Pompe disease: Involvement patterns | Q84625804 |
| | Clinical features and predictors for disease natural progression in adults with Pompe disease: a nationwide prospective observational study | Q21202855 |
| | Recombinant human acid [alpha]-glucosidase: major clinical benefits in infantile-onset Pompe disease | Q28277595 |
| | Validation of a generic approach to muscle water T2 determination at 3T in fat-infiltrated skeletal muscle | Q30769337 |
| | Skeletal muscle quantitative nuclear magnetic resonance imaging follow-up of adult Pompe patients | Q30906462 |
| | A randomized study of alglucosidase alfa in late-onset Pompe's disease | Q34110220 |
| | Differences in the predominance of lysosomal and autophagic pathologies between infants and adults with Pompe disease: implications for therapy | Q34349099 |
| | Pompe disease diagnosis and management guideline. | Q34567528 |
| | Consensus treatment recommendations for late-onset Pompe disease | Q34634354 |
| | Enzyme replacement therapy in late-onset Pompe disease: a systematic literature review | Q34641771 |
| | Survival and associated factors in 268 adults with Pompe disease prior to treatment with enzyme replacement therapy | Q35102705 |
| | The natural course of infantile Pompe's disease: 20 original cases compared with 133 cases from the literature | Q35191008 |
| | Impact of enzyme replacement therapy on survival in adults with Pompe disease: results from a prospective international observational study | Q36758919 |
| | Pompe's disease | Q37302098 |
| | Enzyme replacement therapy in late-onset Pompe's disease: a three-year follow-up | Q39218884 |
| | Characterization of pre- and post-treatment pathology after enzyme replacement therapy for Pompe disease | Q39773493 |
| | Eight years experience with enzyme replacement therapy in two children and one adult with Pompe disease | Q40087057 |
| | alpha-Glucosidase deficiency in generalized glycogenstorage disease (Pompe's disease). | Q41854457 |
| | 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. | Q42629452 |
| | Open-label extension study following the Late-Onset Treatment Study (LOTS) of alglucosidase alfa | Q42670886 |
| | The stability of markers in dried-blood spots for recommended newborn screening disorders in the United States | Q43055719 |
| | A retrospective, multinational, multicenter study on the natural history of infantile-onset Pompe disease | Q44312356 |
| | Disease severity in children and adults with Pompe disease related to age and disease duration | Q47772980 |
| | Observational clinical study in juvenile-adult glycogenosis type 2 patients undergoing enzyme replacement therapy for up to 4 years. | Q50790826 |
| | Effectiveness of enzyme replacement therapy in adults with late-onset Pompe disease: results from the NCS-LSD cohort study. | Q51718537 |
| P433 | issue | 1-2 | |
| P921 | main subject | alglucosidase alfa | Q2919537 |
| P304 | page(s) | 115-123 | |
| P577 | publication date | 2016-05-19 | |
| P1433 | published in | Molecular Genetics and Metabolism | Q6895949 |
| P1476 | title | Prospective exploratory muscle biopsy, imaging, and functional assessment in patients with late-onset Pompe disease treated with alglucosidase alfa: The EMBASSY Study | |
| P478 | volume | 119 | |