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Archives De Pediatrie : Organe Officiel De La Societe Francaise De Pediatrie|March 14, 2024
Evaluation of safety of fluoxetine for cerebellar mutism syndrome in children after posterior fossa surgeryRoxane Varengue, Matthieu Delion, Emilie De Carli, et al.
Pediatric Pulmonology|October 29, 2020
Assessment of respiratory muscles and motor function in children with SMA treated by nusinersenMarta Gómez-García de la Banda, Alessandro Amaddeo, Sonia Khirani, et al.
Neurological Sciences : Official Journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology|September 29, 2022
Use of MFM-20 to monitor SMA types 1 and 2 patients treated with nusinersenLaure Le Goff, Andreea Seferian, Aurelie Phelep, et al.
Biomedicines|March 6, 2021
The lncRNA 44s2 Study Applicability to the Design of 45-55 Exon Skipping Therapeutic Strategy for DMDElena Gargaun, Sestina Falcone, Guilhem Solé, et al.
JAMA Network Open|October 8, 2025
Comparative Clinical Outcomes of Nusinersen and Gene Therapy in Spinal Muscular Atrophy Type 1Juliette Ropars, Claude Cances, Rocio Garcia-Uzquiano, et al.
Frontiers in Pediatrics|March 6, 2020
Palliative Care in SMA Type 1: A Prospective Multicenter French Study Based on Parents' ReportsMarie Hully, Christine Barnerias, Delphine Chabalier, et al.
Annals of Neurology|December 4, 2019
ASC-1 Is a Cell Cycle Regulator Associated with Severe and Mild Forms of MyopathyRocío N Villar-Quiles, Fabio Catervi, Eva Cabet, et al.
Archives De Pediatrie : Organe Officiel De La Societe Francaise De Pediatrie|December 22, 2023
Effect of nusinersen after 3 years of treatment in 57 young children with SMA in terms of SMN2 copy number or typeFrédérique Audic, Sonia M Dubois, Julien Durigneux, et al.
Archives De Pediatrie : Organe Officiel De La Societe Francaise De Pediatrie|January 28, 2025
Evaluation of professional practices in the use of mexiletine for the management of childhood myotonia in French pediatric neuromuscular centers (MEXI-PEDI survey)Sarah Barrière, Véronique Manel, Christine Barnerias, et al.
Orphanet Journal of Rare Diseases|June 14, 2020
Effects of nusinersen after one year of treatment in 123 children with SMA type 1 or 2: a French real-life observational studyFrédérique Audic, Marta Gomez Garcia de la Banda, Delphine Bernoux, et al.
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