Home > Neurology > EAN 2020 > Neuromuscular Disorders > Sustained benefits of avalglucosidase alfa in late-onset Pompe disease

Sustained benefits of avalglucosidase alfa in late-onset Pompe disease

Presented By
Dr Benedikt Schoser, Ludwig-Maximilians-University of Munich, Germany
Conference
EAN 2020
Trial
NEO1; NEO-EXT
The novel investigational enzyme replacement therapy (ERT) avalglucosidase alfa (neoGAA) continued to show clinically meaningful improvement of pulmonary and motor function in patients with late-onset Pompe disease (LOPD) after up to 5.5 years in the NEO1/NEO-EXT studies [1]. NEO-EXT, an ongoing NEO1 extension, assesses efficacy trends of long-term neoGAA use in LOPD patients. In the open-label ascending dose study NEO1, 24 patients enrolled who were either naïve to enzyme replacement therapy (Naïve; n=10) or had received ≥9 months’ standard-of-care alglucosidase alfa (Switch; n=14). They received neoGAA 5, 10, or 20 mg/kg every other week for 6 months. In NEO-EXT, patients continued t...


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