Céline Tard, Françoise Bouhour, Maud Michaud, Stephane Beltran, Maxime Fournier, Florence Demurger, Emmeline Lagrange, Sylvain Nollet, Sabrina Sacconi, Jean-Baptiste Noury, Armelle Magot, Pascal Cintas, Dimitri Renard, Joëlle Deibener-Kaminsky, Claire Lefeuvre, Jean-Baptiste Davion, Emmanuelle Salort-Campana, Azzeddine Arrassi, Nadjib Taouagh, Marco Spinazzi, Shahram Attarian, Pascal Laforêt
INTRODUCTION: Late-onset Pompe disease (LOPD) is characterized by a progressive myopathy resulting from a deficiency of acid α-glucosidase enzyme activity. Enzyme replacement therapy has been shown to be effective, but long-term treatment results vary. Avalglucosidase alfa demonstrated non-inferiority to alglucosidase alfa in a phase 3 study, allowing in France compassionate access for advanced LOPD patients unresponsive to alglucosidase alfa. METHODS: Data from the French Pompe registry were analyzed for patients who benefited from a switch to avalglucosidase alfa with at least 1 year of follow-up...
April 8, 2024: European Journal of Neurology