Long-term Safety and Efficacy of Avalglucosidase Alfa in Patients With Late-Onset Pompe Disease.

Mazen M DimachkieRichard J BarohnBarry ByrneOzlem Goker-AlpanPriya S KishnaniShafeeq LadhaPascal LaforêtKarl Eugen MengelLoren D M PeñaSabrina SacconiVolker StraubJaya TrivediPhilip Van DammeAns T van der PloegJohn VissingPeter YoungKristina An HaackMeredith FosterJane M GilbertPatrick MiossecOlivier VitseTianyue ZhouBenedikt Schosernull null

Published in: Neurology (2022)

Evidence: This study provides Class IV evidence of long-term tolerability and sustained efficacy of avalglucosidase alfa in patients with LOPD after up to 6.5 years.

Keyphrases

late onset
early onset
replacement therapy
open label
clinical trial