| scholarly article | Q13442814 |
| P2093 | author name string | Barry J Byrne | |
| Miguel Sena-Esteves | |||
| Mai K ElMallah | |||
| Allison M Keeler | |||
| Angela L McCall | |||
| Marina Zieger | |||
| Sourav R Choudhury | |||
| Jennifer C Gifford | |||
| Sophia H Todeasa | |||
| Samantha Birsak | |||
| P2860 | cites work | Targeted disruption of the acid alpha-glucosidase gene in mice causes an illness with critical features of both infantile and adult human glycogen storage disease type II | Q28592264 |
| Respiratory function in late-onset Pompe disease patients receiving long-term enzyme replacement therapy for more than 48 months | Q30317955 | ||
| Gel-mediated delivery of AAV1 vectors corrects ventilatory function in Pompe mice with established disease | Q33731154 | ||
| A randomized study of alglucosidase alfa in late-onset Pompe's disease | Q34110220 | ||
| Hydrostatic isolated limb perfusion with adeno-associated virus vectors enhances correction of skeletal muscle in Pompe disease | Q34332408 | ||
| Clinical manifestation and natural course of late-onset Pompe's disease in 54 Dutch patients | Q34554310 | ||
| Pompe disease diagnosis and management guideline. | Q34567528 | ||
| Peripheral nerve and neuromuscular junction pathology in Pompe disease | Q34920690 | ||
| Pompe disease gene therapy. | Q34982843 | ||
| Hypoglossal neuropathology and respiratory activity in pompe mice | Q35084926 | ||
| Comparative impact of AAV and enzyme replacement therapy on respiratory and cardiac function in adult Pompe mice | Q35651103 | ||
| Spinal delivery of AAV vector restores enzyme activity and increases ventilation in Pompe mice | Q35665212 | ||
| Correcting Neuromuscular Deficits With Gene Therapy in Pompe Disease | Q35903220 | ||
| In Vivo Selection Yields AAV-B1 Capsid for Central Nervous System and Muscle Gene Therapy | Q35999976 | ||
| CRIM-negative infantile Pompe disease: characterization of immune responses in patients treated with ERT monotherapy | Q36030998 | ||
| Evaluation of Readministration of a Recombinant Adeno-Associated Virus Vector Expressing Acid Alpha-Glucosidase in Pompe Disease: Preclinical to Clinical Planning | Q36164927 | ||
| The natural course of non-classic Pompe's disease; a review of 225 published cases | Q36245480 | ||
| Copackaged AAV9 Vectors Promote Simultaneous Immune Tolerance and Phenotypic Correction of Pompe Disease | Q36543422 | ||
| Tissue specific promoters improve specificity of AAV9 mediated transgene expression following intra-vascular gene delivery in neonatal mice. | Q36916955 | ||
| Phase I/II trial of adeno-associated virus-mediated alpha-glucosidase gene therapy to the diaphragm for chronic respiratory failure in Pompe disease: initial safety and ventilatory outcomes | Q36945774 | ||
| Intrapleural administration of AAV9 improves neural and cardiorespiratory function in Pompe disease | Q37181416 | ||
| Delayed symptom onset and increased life expectancy in Sandhoff disease mice treated with N-butyldeoxynojirimycin. | Q37209173 | ||
| Neural deficits contribute to respiratory insufficiency in Pompe disease | Q37224150 | ||
| Enhanced efficacy of an AAV vector encoding chimeric, highly secreted acid alpha-glucosidase in glycogen storage disease type II. | Q37259816 | ||
| Temporal neuropathologic and behavioral phenotype of 6neo/6neo Pompe disease mice | Q37345062 | ||
| Pompe disease: a neuromuscular disease with respiratory muscle involvement | Q37364904 | ||
| Sustained correction of motoneuron histopathology following intramuscular delivery of AAV in pompe mice. | Q37696025 | ||
| Low-Dose Liver-Targeted Gene Therapy for Pompe Disease Enhances Therapeutic Efficacy of ERT via Immune Tolerance Induction | Q37717893 | ||
| The respiratory neuromuscular system in Pompe disease | Q38116744 | ||
| Sustained immune tolerance induction in enzyme replacement therapy-treated CRIM-negative patients with infantile Pompe disease | Q38621461 | ||
| Neuropathology in respiratory-related motoneurons in young Pompe (Gaa(-/-)) mice | Q39963807 | ||
| A Neuron-Specific Gene Therapy Relieves Motor Deficits in Pompe Disease Mice. | Q40046102 | ||
| Efficacy of an adeno-associated virus 8-pseudotyped vector in glycogen storage disease type II. | Q40483404 | ||
| Long-term intravenous treatment of Pompe disease with recombinant human alpha-glucosidase from milk | Q40534571 | ||
| Evaluation of respiratory system mechanics in mice using the forced oscillation technique | Q42001774 | ||
| Inspiratory muscle conditioning exercise and diaphragm gene therapy in Pompe disease: Clinical evidence of respiratory plasticity | Q42051051 | ||
| Impaired respiratory function in mdx and mdx/utrn(+/-) mice | Q42741029 | ||
| Oropharyngeal dysphagia in infants and children with infantile Pompe disease. | Q43631261 | ||
| Physiological correction of Pompe disease by systemic delivery of adeno-associated virus serotype 1 vectors | Q45408008 | ||
| Correction of glycogen storage disease type II by an adeno-associated virus vector containing a muscle-specific promoter | Q45457148 | ||
| Correction of the enzymatic and functional deficits in a model of Pompe disease using adeno-associated virus vectors | Q45732679 | ||
| Airway smooth muscle dysfunction in Pompe (Gaa-/- ) mice | Q45866032 | ||
| Rescue of Pompe disease in mice by AAV-mediated liver delivery of secretable acid α-glucosidase. | Q47301525 | ||
| Acute progression of neuromuscular findings in infantile Pompe disease | Q48197007 | ||
| Sleep-disordered breathing and respiratory failure in acid maltase deficiency | Q48682142 | ||
| Delivered dose estimate to standardize airway hyperresponsiveness assessment in mice. | Q51662782 | ||
| Restrictive Lung Disease in the Cu/Zn Superoxide-Dismutase 1 G93A Amyotrophic Lateral Sclerosis Mouse Model. | Q54046876 | ||
| Pompe disease: Design, methodology, and early findings from the Pompe Registry | Q57639968 | ||
| Co-activation of tongue protrudor and retractor muscles during chemoreceptor stimulation in the rat | Q74271358 | ||
| Effect of co-activation of tongue protrudor and retractor muscles on tongue movements and pharyngeal airflow mechanics in the rat | Q78157405 | ||
| A barometric method for measuring ventilation in newborn infants | Q78740607 | ||
| Expanding the phenotype of late-onset Pompe disease: tongue weakness: a new clinical observation | Q85001392 | ||
| P433 | issue | 1 | |
| P407 | language of work or name | English | Q1860 |
| P304 | page(s) | 57-68 | |
| P577 | publication date | 2018-07-25 | |
| P1433 | published in | Human Gene Therapy | Q15757580 |
| P1476 | title | Systemic Delivery of AAVB1-GAA Clears Glycogen and Prolongs Survival in a Mouse Model of Pompe Disease | |
| P478 | volume | 30 |
| Q64039527 | AAV9 gene replacement therapy for respiratory insufficiency in very-long chain acyl-CoA dehydrogenase deficiency |
| Q91655909 | Advancements in AAV-mediated Gene Therapy for Pompe Disease |
| Q92487825 | An emerging phenotype of central nervous system involvement in Pompe disease: from bench to bedside and beyond |
| Q92487836 | Challenges in treating Pompe disease: an industry perspective |
| Q92464614 | Comparisons of Infant and Adult Mice Reveal Age Effects for Liver Depot Gene Therapy in Pompe Disease |
| Q92863328 | Intralingual and Intrapleural AAV Gene Therapy Prolongs Survival in a SOD1 ALS Mouse Model |
| Q92487820 | Liver depot gene therapy for Pompe disease |
| Q90663985 | The Respiratory Phenotype of Pompe Disease Mouse Models |
| Q64257403 | The impact of Pompe disease on smooth muscle: a review |
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