Effect of nusinersen after 3  years of treatment in 57 young children with SMA in terms of SMN2 copy number or type

CONCLUSION: Children with SMA treated with nusinersen continue to make motor acquisitions at 3 years after initiation of treatment. Children with two SMN2 copies had worse motor, respiratory, and orthopedic outcomes after 3 years of treatment than children with three copies.PMID:38135619 | DOI:10.1016/j.arcped.2023.10.009

Source: Archives de Pediatrie - December 22, 2023 Category: Pediatrics Authors: Fr édérique Audic Sonia M Dubois Julien Durigneux Christine Barnerias Arnaud Isapof Marie-Christine Nougues Jean-Baptiste Davion Christian Richelme Carole Vuillerot Laure Legoff Pascal Sabouraud Claude Cances Vincent Laugel Juliette Ropars Caroline Espi Source Type: research