Autophagic myopathies / myofibrillar myopathies / distal myopathies / pompe disease

Long-term safety/efficacy of avalglucosidase alfa is being assessed in LOPD in NEO-EXT (NCT02032524), an ongoing NEO1 (NCT01898364) extension. NEO1 patients (pts), treatment-na ïve (Naïve, n=10) or had received alglucosidase alfa for ≥9mo (Switch, n=14), received avalglucosidase alfa (5, 10, or 20 mg/kg qow) for 6mo; 8 Naïve/11 Switch pts then entered NEO-EXT, continuing their NEO1 dose until switched to 20 mg/kg qow in 2016. Avalglucosidase alfa was generally well-to lerated; the safety profile in NEO-EXT was consistent with NEO1.

Source: Neuromuscular Disorders - September 28, 2020 Category: Neurology Authors: B. Schoser, R. Barohn, B. Byrne, O. Goker-Alpan, P. Kishnani, S. Ladha, P. Laforet, E. Mengel, L. Pena, S. Sacconi, V. Straub, J. Trivedi, P. Van Damme, A. van der Ploeg, J. Vissing, P. Young, K. Haack, I. Ivanina, Y. Wang, M. Dimachkie Source Type: research