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eRAM

encyclopedia of Rare Disease Annotation for Precision Medicine




Disease respiratory failure
Comorbidity C0017921|pompe disease
Sentences 3
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.
PubMedID- 22008916 respiratory failure in pompe disease has been attributed to respiratory muscle dysfunction.
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.

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