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eRAM

encyclopedia of Rare Disease Annotation for Precision Medicine




Disease glycogen storage disease ii
Symptom C1145670|respiratory failure
Sentences 5
PubMedID- 21550241 Recombinant human acid alpha-glucosidase (rhgaa) in adult patients with severe respiratory failure due to pompe disease.
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.
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- 22240224 respiratory failure due to acid maltase deficiency.

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