| P50 | author | Nadine A van der Beek | Q59342385 |
| | Pieter A van Doorn | Q66672518 |
| | Ans van der Ploeg | Q88008512 |
| | Esther Brusse | Q91997266 |
| | Michelle E Kruijshaar | Q125314766 |
| P2093 | author name string | Dimitris Rizopoulos | |
| | Pascal Laforêt | |
| | Aurélie Canal | |
| | Barbara Perniconi | |
| | Nadjib Taouagh | |
| | Jean-Yves Hogrel | |
| | Laurike Harlaar | |
| P2860 | cites work | Effectiveness of enzyme replacement therapy in adults with late-onset Pompe disease: results from the NCS-LSD cohort study. | Q51718537 |
| | Gait deviations in Duchenne muscular dystrophy-Part 1. A systematic review. | Q53276931 |
| | Effect of enzyme replacement therapy on isokinetic strength for all major muscle groups in four patients with Pompe disease—a long-term follow-up | Q57705436 |
| | Clinical trials in Duchenne dystrophy. Standardization and reliability of evaluation procedures | Q70310665 |
| | Muscular weakness assessment: use of normal isometric strength data. The National Isometric Muscle Strength (NIMS) Database Consortium | Q71927227 |
| | Development of a French isometric strength normative database for adults using quantitative muscle testing | Q81380627 |
| | Activity limitations in patients with neuromuscular disorders: a responsiveness study of the ACTIVLIM questionnaire | Q83234964 |
| | High antibody titer in an adult with Pompe disease affects treatment with alglucosidase alfa | Q85000719 |
| | The Rasch-built Pompe-specific activity (R-PAct) scale | Q85807225 |
| | Clinical features and predictors for disease natural progression in adults with Pompe disease: a nationwide prospective observational study | Q21202855 |
| | Intravenous administration of phosphorylated acid alpha-glucosidase leads to uptake of enzyme in heart and skeletal muscle of mice | Q24626527 |
| | Multi-ethnic reference values for spirometry for the 3-95-yr age range: the global lung function 2012 equations | Q28389258 |
| | Standardisation of spirometry | Q29547365 |
| | A randomized study of alglucosidase alfa in late-onset Pompe's disease | Q34110220 |
| | Role of autophagy in the pathogenesis of Pompe disease. | Q34171689 |
| | Enzyme therapy and immune response in relation to CRIM status: the Dutch experience in classic infantile Pompe disease | Q35123163 |
| | Lung MRI and impairment of diaphragmatic function in Pompe disease | Q35595602 |
| | Safety and efficacy of exercise training in adults with Pompe disease: evalution of endurance, muscle strength and core stability before and after a 12 week training program | Q35863802 |
| | Predicted normal values for maximal respiratory pressures in caucasian adults and children | Q36138468 |
| | Effect of enzyme therapy and prognostic factors in 69 adults with Pompe disease: an open-label single-center study | Q36459627 |
| | Neuromuscular ultrasound for evaluation of the diaphragm. | Q36636743 |
| | Impact of enzyme replacement therapy on survival in adults with Pompe disease: results from a prospective international observational study | Q36758919 |
| | Effects of a higher dose of alglucosidase alfa on ventilator-free survival and motor outcome in classic infantile Pompe disease: an open-label single-center study | Q36853458 |
| | Cross-reactive immunologic material status affects treatment outcomes in Pompe disease infants | Q37040458 |
| | Pompe's disease | Q37302098 |
| | Long-term exposure to Myozyme results in a decrease of anti-drug antibodies in late-onset Pompe disease patients | Q37394520 |
| | Polyneuropathy relates to impairment in daily activities, worse gait, and fall-related injuries | Q38752553 |
| | Clinical and pathophysiological clues of respiratory dysfunction in late-onset Pompe disease: New insights from a comparative study by MRI and respiratory function assessment | Q38959711 |
| | European consensus for starting and stopping enzyme replacement therapy in adult patients with Pompe disease: a 10-year experience. | Q39287288 |
| | Characterization of pre- and post-treatment pathology after enzyme replacement therapy for Pompe disease | Q39773493 |
| | Characterization of gait in late onset Pompe disease | Q40537592 |
| | Reference equations for the six-minute walk in healthy adults | Q40835383 |
| | Clinical and molecular aspects of 30 patients with late-onset Pompe disease (LOPD): unusual features and response to treatment | Q41448556 |
| | 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 |
| | Rate of disease progression during long-term follow-up of patients with late-onset Pompe disease. | Q44376591 |
| | Enzyme replacement therapy in the mouse model of Pompe disease | Q44624370 |
| | Long-term benefit of enzyme replacement therapy in Pompe disease: A 5-year prospective study | Q47368096 |
| | Pompe disease in adulthood: effects of antibody formation on enzyme replacement therapy. | Q48179948 |
| | High Sustained Antibody Titers in Patients with Classic Infantile Pompe Disease Following Immunomodulation at Start of Enzyme Replacement Therapy | Q50083948 |
| | Observational clinical study in juvenile-adult glycogenosis type 2 patients undergoing enzyme replacement therapy for up to 4 years. | Q50790826 |
| | Observational clinical study of 22 adult-onset Pompe disease patients undergoing enzyme replacement therapy over 5years. | Q51547323 |
| | ATS/ERS Statement on respiratory muscle testing. | Q51706601 |
| P433 | issue | 19 | |
| P304 | page(s) | e1756-e1767 | |
| P577 | publication date | 2019-10-16 | |
| P1433 | published in | Neurology | Q1161692 |
| P1476 | title | Large variation in effects during 10 years of enzyme therapy in adults with Pompe disease | |
| P478 | volume | 93 | |