Marta Gómez García de la Banda

Effects of nusinersen after one year of treatment in 123 children with SMA type 1 or 2: a French real-life observational study

Frédérique Audic, Marta Gómez García de la Banda, Delphine Bernoux et al. · Orphanet Journal of Rare Diseases · 2020 · 101 citations · Full text

Medicine, Neurology, Pediatrics +17

Safety and efficacy of apitegromab in nonambulatory type 2 or type 3 spinal muscular atrophy (SAPPHIRE): a phase 3, double-blind, randomised, placebo-controlled trial

Thomas O. Crawford, Eugenio Mercuri, Heike Kölbel et al. · The Lancet Neurology · 2025 · 19 citations · Full text

Medicine, Neurology, Pharmacotherapy +5

Respiratory management of spinal muscular atrophy type 1 patients treated with Nusinersen

Joris Menard, A. Seferian, Emmanuelle Fleurence et al. · Pediatric Pulmonology · 2022 · 17 citations

Medicine, Clinical Sciences, Pediatrics +16

Diagnostic interest of whole-body MRI in early- and late-onset LAMA2 muscular dystrophies: a large international cohort

Susana Quijano‐Roy, Jana Haberlová, Claudia Castiglioni et al. · Journal of Neurology · 2021 · 13 citations · Full text

Medicine, Neurology, Radiology +14

NTRK1 gene-related congenital insensitivity to pain with anhidrosis: a nationwide multicenter retrospective study

Andoni Echaniz‐Laguna, Cécilia Altuzarra, Alain Verloès et al. · Neurogenetics · 2021 · 12 citations

Life Sciences, Neuroscience, Human Genetics +7