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Papadimas GK, Anagnostopoulos C, Xirou S, Michelakakis H, Terzis G, Mavridou I, et al. Effect of long term enzyme replacement therapy in late onset Pompe disease: a single-centre experience. Neuromuscul Disord. 2021;31(2):91–100.

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•• Schoser B, Roberts M, Byrne BJ, Sitaraman S, Jiang H, Laforêt P, et al. Safety and efficacy of cipaglucosidase alfa plus miglustat versus alglucosidase alfa plus placebo in late-onset Pompe disease (PROPEL): an international, randomised, double-blind, parallel-group, phase 3 trial. Lancet Neurol. 2021;20(12):1027–37. This paper reports the results of the Phase 3 randomized controlled trial of cipaglucosidase alfa in combination with miglustat, a stabilizer of the enzyme, vs. alglucosidase alfa and a placebo in treatment naive patients with LOPD. This was comparison of cipaglucosidase alfa, a newly designed ERT with through clonal selection of rhGAA with CHO-cell derived M6P and bis-M6P moieties, to alglucosidase alfa, the standard of care in LOPD since 2010. The results show that avalglucosidase alfa was non-inferior to alglucosidase alfa, but there were trends in some outcome measures that showed cipaglucosidase alfa to be significantly better than alglucosidase alfa. This new drug will go up for FDA review in Fall 2022.

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• Zhou Z, Austin GL, Shaffer R, Armstrong DD, Gentry MS. Antibody-mediated enzyme therapeutics and applications in glycogen storage diseases. Trends Mol Med. 2019;25(12):1094–109. A good review of new efforts to develop enzyme replacement therapies conjugated with monoclonal antibodies to allow better delivery to skeletal muscle and cardiac tissue.

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