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1385 news items
Research
Allele-specific gene silencing by RNA interference: a robust, safe and effective treatment for dominant hereditary diseases
On 17/12/2025
Allele-specific gene silencing by RNA interference is a promising therapeutic approach for dominant hereditary diseases. This strategy is based on the targeted inhibition of messenger…
Research
Psychological evaluations to improve NIV compliance in Steinert disease
On 05/12/2025
An observational study evaluated factors influencing compliance with the initiation of non-invasive ventilation (NIV) treatment for sleep-disordered breathing in 13 adults with myotonic dystrophy type…
Research
Pierre Klein awarded the Impulsion Award at the 2025 JSFM
On 04/12/2025
Pierre Klein is a senior postdoctoral researcher at the Centre of Research in Myology in the Repeat Expansions & Myotonic Dystrophy (REDs) team led by Denis…
Research
Gene therapy for SMA, available in the United States for patients of any age
On 03/12/2025
A new indication for gene therapy in proximal spinal muscular atrophy (SMA), involving a single intrathecal injection of Itvisma, has just been approved by the…
Research
Looking back at 2025 SFM Annual Meeting
On 02/12/2025
Nearly 400 participants gathered at the 2025 French Society of Myology Conference (JSFM), which took place from 19 to 21 November 2025 in Aix-Les-Bains. Focus…
Research
Few swallowing disorders in children with SMA treated at a presymptomatic phase
On 01/12/2025
A consortium of clinicians from the United States, New Zealand and Brazil reports the results of a study aimed at investigating bulbar function in infants…
Research
A new gene linked to excitation-contraction coupling responsible for early-onset myopathy
On 26/11/2025
Spanish researchers report clinical and biological data from two unrelated consanguineous families in which a new gene for early-onset myopathy has been identified: the first…
Research
An original mode of administration in the treatment of refractory myasthenia gravis
On 24/11/2025
An international consortium of clinicians reports the results of a phase III open-label trial designed to assess the benefit and tolerability of a monoclonal antibody,…
Research
More is now known about the pathophysiology of myotubularin
On 21/11/2025
The role of myotubularin, a muscle protein involved in X-linked recessive tubular myopathy, remains poorly understood. Researchers in Strasbourg and Toulouse report on their work…
Research
The lysosome, a potential therapeutic target in DMD
On 20/11/2025
Researchers from the Institute of Myology and Généthon report pioneering work in the treatment of Duchenne muscular dystrophy (DMD): they started from the observation that…
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…
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…
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…