PedAM
Pediatric Disease Annotations & Medicines
| Disease | respiratory failure |
| Phenotype | C0017921|pompe disease |
| Sentences | 4 |
| PubMedID- 23570273 | 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. |
| PubMedID- 22008916 | respiratory failure in pompe disease has been attributed to respiratory muscle dysfunction. |
| PubMedID- 23531252 | Because most adult patients with pompe disease eventually die of respiratory failure [1,7], the beneficial effect of ert on survival is likely to be related to its positive effect on pulmonary function. |
| PubMedID- 24399863 | respiratory failure in patients with pompe disease can range from insidious to acute onset and respiratory muscle involvement is the most common cause of early death in these patients. |
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