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2414 news items
Research
Preclinical data for a drug candidate in Steinert disease
On 19/11/2025
A preclinical study evaluated SAR446268, an artificial microRNA produced by an AAV vector and targeting DMPK (dystrophin myotonic protein kinase) RNA designed by Sanofi, in…
Research
Valentin Henriet awarded the ‘Best Technical Poster’ prize at the MYO-MRI+ 2025 conference
On 17/11/2025
Valentin Henriet is a PhD student in the Nuclear Magnetic Resonance (NMR) Imaging and Spectroscopy Laboratory. The poster entitled ‘Creatine-CEST-based pH mapping in healthy volunteer…
Events
M&M’s – Muscle Monday Seminar – 17 nov. – Dr Mario Amendola (France)
On 17/11/2025
Gene Editing for DMD Treatment Monday 17 November – from noon to 1pm (Paris time) Dr Mario AMENDOLA (Genethon, Paris, France) On prior registration…
Research
The Institute is developing an app using AI to assess motor function
On 14/11/2025
Romain Feigean is a researcher at the Neuromuscular Physiology and Evaluation Laboratory headed by Jean-Yves Hogrel at the Institute’s NIC. He is particularly involved in…
Research
Ways to avoid liver toxicity from AAV
On 14/11/2025
Researchers at the Regenxbio pharmaceutical laboratory and American academics review the hepatic toxicity of adeno-associated viruses (AAV) in the context of human gene therapy, and…
Events
ERN EURO-NMD webinar, 20 Nov.: Prof. Valentina Damato (Italy)
On 14/11/2025
Update on antibodies in Neuromuscular Junction Disorders Thursday 20 November 2025 – 16:00 – 17:00 Paris time Prof. Valentina Damato (University of Florence, Italy) >…
Research
Towards better reuse of patient data in DMD
On 13/11/2025
A consortium bringing together patient associations, clinicians and methodologists from several countries reports on the FAIR project, which aims to better coordinate and standardise registries…
Research
A standardised digital platform for Charcot-Marie-Tooth disease
On 12/11/2025
American and Australian researchers have developed a digital platform designed to facilitate the assessment, even remotely, of patients with Charcot-Marie-Tooth disease (CMT): the platform is…
Research
A genetic predisposition to muscle toxicity from checkpoint inhibitors?
On 10/11/2025
Checkpoint inhibitors used in cancer treatment are likely to cause inflammatory myopathies and/or myocarditis. German and Swiss clinicians sought to identify risk factors for the…
Research
A genetic isolate of limb-girdle muscular dystrophy in Uruguay
On 10/11/2025
Clinicians and researchers in Montevideo, Uruguay, report clinical and biological data from the world’s largest cohort of autosomal dominant limb-girdle muscular dystrophy linked to the…
Research
Gene therapy appears to be more favourable than nusinersen in symptomatic type I SMA, according to an initial comparative analysis by the French SMA Registry.
On 07/11/2025
Based on data from 309 patients listed in the SMA France Registry and suffering from type I proximal spinal muscular atrophy (SMA), French clinicians conducted…
Research
A new familial case of distal myopathy linked to the STPAN1 gene
On 07/11/2025
Belgian clinicians report clinical and biological data from a family diagnosed with distal myopathy linked to the STPAN1 gene: the clinical picture consisted of muscle…
Events
ERN EURO-NMD webinar, 6 Nov.: Dr. Lorenzo Maggi (Italy) & Lutgarde Allard (EuMGA & Patient expert)
On 06/11/2025
How to evaluate patients with Myasthenia Gravis Thursday 6 November 2025 – 16:00 – 17:00 Paris time Dr. Lorenzo Maggi (Fondazione IRCCS Istituto Neurologico Carlo…
Research
A mitochondrial gene responsible for exercise intolerance and rhabdomyolysis
On 06/11/2025
Myologists from the Institut de Myologie (clinicians from the Service of Neuro-Myology and researchers from the CRM and the Morphology Unit) and the Cochin Hospital…
Research
A useful electrophysiological score in gene therapy for SMA
On 06/11/2025
French clinicians report their experience with measuring compound muscle action potentials (CMAP) in infants with symptomatic SMA who have received gene therapy (onasemnogene abeparvovec): 19…
Events
M&M’s – Muscle Monday Seminar – 10 Nov. – Dr Anna Urciuolo (Italy)
On 05/11/2025
Advanced organoids for studying the human neuromuscular system in vitro Monday 10 November – from 12 to 1pm (Paris time) Dr Anna URCIUOLO (Italy) invited…
Research
The use of CAR-T cells to support gene therapy in DMD
On 05/11/2025
Researchers from Généthon (Evry) used genetically modified T lymphocytes (FAP-type CAR-T) to improve gene-drug transfers via adeno-associated viruses (AAV) in gene therapy: after two injections,…
Events
Focus on I-Motion clinical trial platforms
On 04/11/2025
Interview with Dr Marina Colella, Deputy Director and Head of Medical Staff at I-Motion, and Saadane Kirouani, Deputy Director and Head of Non-Medical Staff. What…
Research
Changes in respiratory function in adults with SMA treated with nusinersen
On 04/11/2025
German clinicians studied the respiratory parameters of 192 adult patients with SMA receiving nusinersen treatment: patients had been treated for an average of 3.2 years,…
Research
A French study on the muscular and cardiac toxicity of immune checkpoint inhibitors
On 03/11/2025
French researchers have investigated the adverse effects of immune checkpoint inhibitors, which are latest-generation anti-cancer drugs: their research focused on the myotoxic and cardiotoxic effects…