Claude Cances
Toulouse, France
Last Updated: 09/26/2026
Save publications for later
Sign Up
Not sure about your diagnosis?
Check Your Symptoms
Found 89 publications
TTC19-related mitochondrial disease: A characteristic neuroimaging signature?
Journal: Molecular genetics and metabolism
Published: June 02, 2026
Author Correction: Intrathecal onasemnogene abeparvovec for treatment-experienced patients with spinal muscular atrophy: a phase 3b, open-label trial.
Journal: Nature medicine
Published: January 28, 2026
A plain language summary of the SAPPHIRE clinical trial of apitegromab in children and young adults with spinal muscular atrophy.
Journal: Expert review of neurotherapeutics
Published: November 13, 2025
Comparative Clinical Outcomes of Nusinersen and Gene Therapy in Spinal Muscular Atrophy Type 1.
Journal: JAMA network open
Published: October 08, 2025
Phenotype-specific muscle proteomic profiling in titinopathies.
Journal: Acta neuropathologica communications
Published: September 12, 2025
Intrathecal onasemnogene abeparvovec for treatment-experienced patients with spinal muscular atrophy: a phase 3b, open-label trial.
Journal: Nature medicine
Published: July 23, 2025
REGISTRE SMA FRANCE: A nationwide observational registry of patients with spinal muscular atrophy in France.
Journal: Journal of neuromuscular diseases
Published: July 08, 2025
Prevalence of pediatric neuromuscular disorders in the Southwest region of France.
Journal: Neuromuscular disorders : NMD
Published: May 27, 2025
Innovative treatments of pediatric spinal muscular atrophy: The decision-making process in France.
Journal: European journal of medical genetics
Published: May 16, 2025
Diagnostic Impasse and Wandering in Patients With Rare Neuromuscular Diseases: Insights Into Patient Characteristics From the French National Network for Rare Neuromuscular Diseases (FILNEMUS) and the French National Rare Disease Database (BNDMR).
Journal: European journal of neurology
Published: April 03, 2025
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.
Journal: The Lancet. Neurology
Published: March 25, 2025
Last Updated: 09/26/2026