Transplantační medicína

Efficacy of avalglucosidase alfa on forced vital capacity percent predicted in treatment-naïve patients with late-onset Pompe disease
^{Mozaffar T, et al. Mol Genet Metab Rep. 2024;40:101109.}

Presentación de apoyo visual utilizada en evento Sanofi acerca de "ISPAD 2024: Nuevas Perspectivas" a partir de un resumen de los temas destacados del Congreso ISPAD 2024. Confeccionado por las doctoras: Liliana Trifone, Carolina Martinez Mateu, Adriana Roussos, Marcela Raggio y Florencia Grabois.

Clinical insight meets scientific innovation to develop a next generation ERT for Pompe disease
^{Kishnani PS, et al. Mol Genet Metab. 2024;143(1-2):108559.}

Safety and Pharmacokinetics of Nirsevimab in Immunocompromised Children
Domachowske J, Hamrén UW, Banu I, Baronio R, Basavaraju B, Koen A, Leach A, Mankad VS, Pannaraj PS, Soler-Palacin P, Takas T, Mori M, Villafana T,  for the MUSIC Study Group.

ALPROLIX has been studied across a range of trials in both adults and children. Here you can read an overview of the publications and access links to the online manuscripts.