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The clinical relevance of outcomes used in late-onset Pompe disease: can we do better?

Pompe disease/glycogen storage disease type II, is a rare, lysosomal storage disorder associated with progressive proximal myopathy, causing a gradual loss of muscular function and respiratory insufficiency. Studies of patients with late-onset Pompe disease have used endpoints such as the 6-minute w...

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Bibliografiska uppgifter
Huvudupphovsmän: Lachmann, Robin, Schoser, Benedikt
Materialtyp: Artigo
Språk:Inglês
Publicerad: BioMed Central 2013
Ämnen:
Länkar:https://ncbi.nlm.nih.gov/pmc/articles/PMC4015278/
https://ncbi.nlm.nih.gov/pubmed/24119230
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/1750-1172-8-160
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