| scholarly article | Q13442814 |
| P2093 | author name string | David D Fuller | |
| Barry J Byrne | |||
| Darin J Falk | |||
| Paul J Reier | |||
| Kai Qiu | |||
| P2860 | cites work | Pompe disease: Design, methodology, and early findings from the Pompe Registry | Q57639968 |
| [Glycogenesis Type II (M. Pompe). Selective failure of the respiratory musculature--a rare first symptom] | Q64047278 | ||
| Generalized glycogen storage disease in Japanese quail (Coturnix coturnix japonica) | Q70909991 | ||
| Pompe's disease | Q70982390 | ||
| [Three hypotonic neonates with hypertrophic cardiomyopathy: Pompe's disease] | Q77344859 | ||
| A barometric method for measuring ventilation in newborn infants | Q78740607 | ||
| Clinical and metabolic correction of pompe disease by enzyme therapy in acid maltase-deficient quail | Q24564823 | ||
| Recombinant human acid [alpha]-glucosidase: major clinical benefits in infantile-onset Pompe disease | Q28277595 | ||
| Rescue of enzyme deficiency in embryonic diaphragm in a mouse model of metabolic myopathy: Pompe disease | Q28508908 | ||
| 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 | ||
| Gel-mediated delivery of AAV1 vectors corrects ventilatory function in Pompe mice with established disease | Q33731154 | ||
| Recombinant adeno-associated virus vector: use for transgene expression and anterograde tract tracing in the CNS | Q34472181 | ||
| Acid alpha-glucosidase deficiency (glycogenosis type II, Pompe disease). | Q34597228 | ||
| Hypoglossal neuropathology and respiratory activity in pompe mice | Q35084926 | ||
| Glycogen disease of skeletal muscle; report of two cases and review of literature | Q35531389 | ||
| Pompe disease in infants and children | Q35764507 | ||
| Glycogen disease resembling mongolism, cretinism, and amytonia congenita; case report and review of literature | Q35898188 | ||
| Recombinant adeno-associated viral vectors as therapeutic agents to treat neurological disorders. | Q36368045 | ||
| Cervical prephrenic interneurons in the normal and lesioned spinal cord of the adult rat | Q37003791 | ||
| Cardiac remodeling after enzyme replacement therapy with acid alpha-glucosidase for infants with Pompe disease | Q37196681 | ||
| Neural deficits contribute to respiratory insufficiency in Pompe disease | Q37224150 | ||
| Temporal neuropathologic and behavioral phenotype of 6neo/6neo Pompe disease mice | Q37345062 | ||
| Therapy for lysosomal storage disorders | Q37656018 | ||
| Long-term intravenous treatment of Pompe disease with recombinant human alpha-glucosidase from milk | Q40534571 | ||
| Intractable fever and cortical neuronal glycogen storage in glycogenosis type 2. | Q40596816 | ||
| Production and purification of serotype 1, 2, and 5 recombinant adeno-associated viral vectors | Q40692830 | ||
| The phrenic motor nucleus in the adult mouse. | Q42030823 | ||
| Oropharyngeal dysphagia in infants and children with infantile Pompe disease. | Q43631261 | ||
| Respiratory insufficiency and limb muscle weakness in adults with Pompe's disease | Q44306148 | ||
| Nervous system in Pompe's disease. Ultrastructure and biochemistry | Q44576666 | ||
| Enzyme replacement therapy in the mouse model of Pompe disease | Q44624370 | ||
| Recombinant AAV viral vectors pseudotyped with viral capsids from serotypes 1, 2, and 5 display differential efficiency and cell tropism after delivery to different regions of the central nervous system | Q45007196 | ||
| Physiological correction of Pompe disease by systemic delivery of adeno-associated virus serotype 1 vectors | Q45408008 | ||
| Correction of the enzymatic and functional deficits in a model of Pompe disease using adeno-associated virus vectors | Q45732679 | ||
| Efficient transduction of green fluorescent protein in spinal cord neurons using adeno-associated virus vectors containing cell type-specific promoters | Q45764253 | ||
| Long-term clinical improvement in MPTP-lesioned primates after gene therapy with AAV-hAADC. | Q45859568 | ||
| Acute progression of neuromuscular findings in infantile Pompe disease | Q48197007 | ||
| Pompe's disease: an inborn lysosomal disorder with storage of glycogen. A study of brain and striated muscle | Q48681355 | ||
| A correlated histochemical and quantitative study on cerebral glycogen after brain injury in the rat | Q49125271 | ||
| CRIM-negative infantile Pompe disease: 42-month treatment outcome. | Q50436382 | ||
| POMPE'S DISEASE (DIFFUSE GLYCOGENOSIS) WITH NEURONAL STORAGE | Q51260553 | ||
| Use of the muscle volume analyzer to evaluate enzyme replacement therapy in late-onset Pompe disease. | Q51604035 | ||
| Infantile-onset glycogen storage disease type II (Pompe disease): report of a case with genetic diagnosis and pathological findings. | Q51937484 | ||
| P433 | issue | 1 | |
| P304 | page(s) | 21-27 | |
| P577 | publication date | 2011-10-18 | |
| P1433 | published in | Molecular Therapy | Q15762400 |
| P1476 | title | Spinal delivery of AAV vector restores enzyme activity and increases ventilation in Pompe mice | |
| P478 | volume | 20 |
| Q47564638 | AAV gene delivery to the spinal cord: serotypes, methods, candidate diseases, and clinical trials |
| 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 |
| Q37694653 | B-Cell depletion and immunomodulation before initiation of enzyme replacement therapy blocks the immune response to acid alpha-glucosidase in infantile-onset Pompe disease. |
| Q38834554 | Combination Therapies for Lysosomal Storage Diseases: A Complex Answer to a Simple Problem |
| Q35651103 | Comparative impact of AAV and enzyme replacement therapy on respiratory and cardiac function in adult Pompe mice |
| Q35903220 | Correcting Neuromuscular Deficits With Gene Therapy in Pompe Disease |
| Q36945130 | Diaphragm and ventilatory dysfunction during cancer cachexia |
| Q91933156 | Dynamic respiratory muscle function in late-onset Pompe disease |
| Q38788613 | Histopathological Evaluation of Skeletal Muscle with Specific Reference to Mouse Models of Muscular Dystrophy. |
| Q37181416 | Intrapleural administration of AAV9 improves neural and cardiorespiratory function in Pompe disease |
| Q64244491 | Intravenous Injection of an AAV-PHP.B Vector Encoding Human Acid α-Glucosidase Rescues Both Muscle and CNS Defects in Murine Pompe Disease |
| Q41625013 | Long-term neurologic and cardiac correction by intrathecal gene therapy in Pompe disease. |
| Q92487832 | Pompe disease gene therapy: neural manifestations require consideration of CNS directed therapy |
| Q35029971 | Pompe disease: literature review and case series |
| Q92487813 | Progress and challenges of gene therapy for Pompe disease |
| Q33586940 | Retrograde gene delivery to hypoglossal motoneurons using adeno-associated virus serotype 9. |
| Q59358757 | Systemic Delivery of AAVB1-GAA Clears Glycogen and Prolongs Survival in a Mouse Model of Pompe Disease |
| Q26767363 | Targeted approaches to induce immune tolerance for Pompe disease therapy |
| Q34749792 | Targeted delivery of TrkB receptor to phrenic motoneurons enhances functional recovery of rhythmic phrenic activity after cervical spinal hemisection. |
| Q90894134 | Targeted intraspinal injections to assess therapies in rodent models of neurological disorders |
| Q90663985 | The Respiratory Phenotype of Pompe Disease Mouse Models |
| Q38116744 | The respiratory neuromuscular system in Pompe disease |
| Q39404266 | Transcriptome assessment of the Pompe (Gaa-/-) mouse spinal cord indicates widespread neuropathology. |
Search more.