36-Months follow-up assessment after cessation and resuming of enzyme replacement therapy in late onset Pompe disease: data from the Swiss Pompe Registry
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DOI: 10.1007/s00415-018-9065-7
Abstract: IntroductionAlthough not curative, enzyme replacement therapy (ERT) with recombinant human acid alpha-glucosidase enzyme has shown to be effective in the treatment of late-onset Pompe disease (LOPD). For this potentially life-long treatment, little is known on… read more here.
Keywords: replacement therapy; late onset; enzyme replacement; resuming ert ... See more keywords