| scholarly article | Q13442814 |
| P50 | author | Lingli Zhang | Q92498089 |
| P2093 | author name string | Min Chen | |
| Shuyan Quan | |||
| P2860 | cites work | Brain development in infantile-onset Pompe disease treated by enzyme replacement therapy. | Q48466555 |
| Dysfunction of endocytic and autophagic pathways in a lysosomal storage disease. | Q50736816 | ||
| Safety and efficacy of alternative alglucosidase alfa regimens in Pompe disease. | Q51015079 | ||
| Clinical outcomes after long-term treatment with alglucosidase alfa in infants and children with advanced Pompe disease. | Q51768443 | ||
| Recombinant human alpha-glucosidase from rabbit milk in Pompe patients. | Q55034196 | ||
| Left Ventricular Geometry, Global Function, and Dyssynchrony in Infants and Children With Pompe Cardiomyopathy Undergoing Enzyme Replacement Therapy | Q61717681 | ||
| Comparison of maltose and acarbose as inhibitors of maltase-glucoamylase activity in assaying acid alpha-glucosidase activity in dried blood spots for the diagnosis of infantile Pompe disease | Q83350397 | ||
| Ambulatory electrocardiogram analysis in infants treated with recombinant human acid alpha-glucosidase enzyme replacement therapy for Pompe disease | Q83350412 | ||
| Identification of two subtypes of infantile acid maltase deficiency | Q22254747 | ||
| Enzyme replacement therapy for infantile-onset Pompe disease | Q24187658 | ||
| Recombinant human acid [alpha]-glucosidase: major clinical benefits in infantile-onset Pompe disease | Q28277595 | ||
| Durable and sustained immune tolerance to ERT in Pompe disease with entrenched immune responses | Q28595509 | ||
| GRADE guidelines: 1. Introduction-GRADE evidence profiles and summary of findings tables | Q29547885 | ||
| Chinese hamster ovary cell-derived recombinant human acid alpha-glucosidase in infantile-onset Pompe disease | Q30489156 | ||
| Recombinant human acid alpha-glucosidase enzyme therapy for infantile glycogen storage disease type II: results of a phase I/II clinical trial | Q33941289 | ||
| Update of the Pompe disease mutation database with 107 sequence variants and a format for severity rating | Q34011226 | ||
| Pompe disease diagnosis and management guideline. | Q34567528 | ||
| The impact of antibodies on clinical outcomes in diseases treated with therapeutic protein: lessons learned from infantile Pompe disease | Q34629805 | ||
| Early treatment with alglucosidase alpha prolongs long-term survival of infants with Pompe disease | Q35087282 | ||
| Enzyme therapy and immune response in relation to CRIM status: the Dutch experience in classic infantile Pompe disease | Q35123163 | ||
| The natural course of infantile Pompe's disease: 20 original cases compared with 133 cases from the literature | Q35191008 | ||
| CRIM-negative infantile Pompe disease: characterization of immune responses in patients treated with ERT monotherapy | Q36030998 | ||
| Lysosomal dysfunction, cellular pathology and clinical symptoms: basic principles | Q36486796 | ||
| Enhanced response to enzyme replacement therapy in Pompe disease after the induction of immune tolerance | Q36492202 | ||
| Skeletal muscle pathology of infantile Pompe disease during long-term enzyme replacement therapy | Q36953239 | ||
| Successful immune tolerance induction to enzyme replacement therapy in CRIM-negative infantile Pompe disease. | Q37012107 | ||
| Cardiac remodeling after enzyme replacement therapy with acid alpha-glucosidase for infants with Pompe disease | Q37196681 | ||
| Successful Desensitisation in a Patient with CRIM-Positive Infantile-Onset Pompe Disease. | Q37500689 | ||
| Pompe disease: early diagnosis and early treatment make a difference. | Q38103090 | ||
| CRIM-Negative Pompe Disease Patients with Satisfactory Clinical Outcomes on Enzyme Replacement Therapy | Q42183681 | ||
| Safety and efficacy of recombinant acid alpha-glucosidase (rhGAA) in patients with classical infantile Pompe disease: results of a phase II clinical trial | Q47869064 | ||
| Treatment of infantile Pompe disease with alglucosidase alpha: the UK experience | Q48324978 | ||
| P921 | main subject | enzyme replacement therapy | Q916766 |
| P304 | page(s) | CD011539 | |
| P577 | publication date | 2017-11-20 | |
| P1433 | published in | Cochrane Database of Systematic Reviews | Q15750361 |
| P1476 | title | Enzyme replacement therapy for infantile-onset Pompe disease | |
| P478 | volume | 11 |
| Q57798439 | Clinical and molecular characterization of Korean children with infantile and late-onset Pompe disease: 10 years of experience with enzyme replacement therapy at a single center |
| Q92787885 | Comprehensive approach to weaning in difficult-to-wean infantile and juvenile-onset glycogen-storage disease type II patients: a case series |
| Q92549228 | Considerations for evaluating the effectiveness and long-term outcome of enzyme replacement therapy in Pompe disease |
| Q90598081 | Improvement in Cardiac Function With Enzyme Replacement Therapy in a Patient With Infantile-Onset Pompe Disease |
| Q92487791 | Long-term outcome and unmet needs in infantile-onset Pompe disease |
| Q59358387 | Molecular Therapies for Muscular Dystrophies |
| Q89920152 | Neuropathophysiology of Lysosomal Storage Diseases: Synaptic Dysfunction as a Starting Point for Disease Progression |
| Q92487841 | Pompe disease: what are we missing? |
| Q58565558 | Storage diseases with hypertrophic cardiomyopathy phenotype |
| Q64257403 | The impact of Pompe disease on smooth muscle: a review |
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