Laure Le Goff

Natural history of Type 1 spinal muscular atrophy: a retrospective, global, multicenter study

Claude Cancés, D. Vlodavets, Riccardo Masson et al. · Orphanet Journal of Rare Diseases · 2022 · 35 citations · Full text

Medicine, Neurology, Neuroscience +16

Assessment of the validity and reliability of the 32-item Motor Function Measure in individuals with Type 2 or non-ambulant Type 3 spinal muscular atrophy

Dylan Trundell, Stephanie Le Scouiller, Laure Le Goff et al. · PLoS ONE · 2020 · 23 citations · Full text

Medicine, Neurology, Rehabilitation Medicine +16

Real-world multidisciplinary outcomes of onasemnogene abeparvovec monotherapy in patients with spinal muscular atrophy type 1: experience of the French cohort in the first three years of treatment

Isabelle Desguerre, Rémi Barrois, Frédérique Audic et al. · Orphanet Journal of Rare Diseases · 2024 · 18 citations · Full text

Medicine, Neurology, Pharmacotherapy +17

Responsiveness and Minimal Clinically Important Difference of the Motor Function Measure in Collagen VI-Related Dystrophies and Laminin Alpha2-Related Muscular Dystrophy

Laure Le Goff, Katherine G. Meilleur, Gina Norato et al. · Archives of Physical Medicine and Rehabilitation · 2020 · 16 citations · Full text

Medicine, Neurology, Functional Movement Assessment +16

Severe congenital myasthenic syndromes caused by agrin mutations affecting secretion by motoneurons

Arnaud Jacquier, Valérie Risson, Thomas Simonet et al. · Acta Neuropathologica · 2022 · 14 citations · Full text

Life Sciences, Genetics, Neuroscience +14