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Real-life Effectiveness 1 Year after Switching to Avalglucosidase Alfa in Late-Onset Pompe Disease Patients Worsening on Alglucosidase Alfa Therapy: A French Cohort Study

Celine Tard,Francoise Bouhour,Maud Michaud,Stephane Beltran, Maxime Fournier,Florence Demurger,Emmeline Lagrange,Sylvain Nollet,Sabrina Sacconi,Jean-Baptiste Noury,Armelle Magot,Pascal Cintas,Dimitri Renard,Joelle Deibener-Kaminsky,Claire Lefeuvre,Jean-Baptiste Davion,Emmanuelle Salort-Campana,Azzeddine Arrassi,Nadjib Taouagh,Marco Spinazzi,Shahram Attarian,Pascal Laforet

European Journal of Neurology（2024）

Cited 1|Views36
Key words
enzymotherapy,glycogenosis,switch,treatment management,unmet needs
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