Recombinant human acid alpha-glucosidase (rhGAA) in adult patients with severe respiratory failure due to Pompe disease.
Neuromuscular Disorders(2011)
摘要
Pompe disease is a rare metabolic myopathy caused by lysosomal α-glucosidase deficiency. Pompe disease ranges from a rapidly progressive course when symptoms present in infancy to a more slowly progressive rate when symptoms present in childhood or adulthood.
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关键词
Pompe disease,Alglucosidase alfa,Enzyme replacement therapy,rhGAA
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