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Improvement of bone mineral density after enzyme replacement therapy in Chinese late-onset Pompe disease patients

OBJECTIVE: Late-onset Pompe disease (LOPD) is a lysosomal storage disease resulted from deficiency of the enzyme acid α-glucosidase. Patients usually develop a limb-girdle pattern of myopathy and respiratory impairment, and enzyme replacement therapy (ERT) is the only specific treatment available. R...

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Pubblicato in:BMC Res Notes
Autori principali: Sheng, Bun, Chu, Yim Pui, Wong, Wa Tai, Yau, Eric Kin Cheong, Chen, Sammy Pak Lam, Luk, Wing Hang
Natura: Artigo
Lingua:Inglês
Pubblicazione: BioMed Central 2017
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Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC5534128/
https://ncbi.nlm.nih.gov/pubmed/28754168
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s13104-017-2681-y
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