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1385 news items
Research
Chemical inhibition of SUMOylation activates the FSHD locus
On 26/03/2026
Researchers in Marseille, in collaboration with a researcher from the Institute of Myology*, have investigated the epigenetic regulation of DUX4 expression—the protein responsible for muscle…
Research
Improved efficacy of ERT in pediatric forms of Pompe disease due to newborn screening
On 26/03/2026
A growing number of countries, including the United States, have implemented newborn screening for Pompe disease. American clinicians review the cases of children screened and…
Research
The use of autologous CD19 CAR-T cells in refractory autoimmune diseases
On 25/03/2026
A European consortium of researchers reviews the therapeutic use of genetically modified autologous T cells (CAR-T cells): this autologous cell therapy was administered to patients…
Research
The sarcoglycanopathies pattern in whole-body muscle imaging
On 24/03/2026
An international research consortium has reported magnetic resonance imaging (MRI) data (whole-body MRI in this case) from 64 patients diagnosed with sarcoglycanopathy: 43 had LGMD-R3…
Research
An unusual presentation of congenital myasthenic syndrome
On 20/03/2026
Clinicians in Hong Kong report an unusual case of congenital myasthenic syndrome (CMS) that initially suggested an eating disorder: the patient was 42 years old…
Research
TK2 deficiency: from the treatment of early-onset forms to understanding the natural history of late-onset forms
On 13/03/2026
In January 2026, the European Medicines Agency (EMA) issued a favourable opinion on granting marketing authorisation (MA) for Kygevvi (doxecitine and doxribtimine) for the treatment…
Research
An increased risk of urinary disorders in cases of autoimmune myasthenia
On 10/03/2026
In order to study immune disorders in autoimmune myasthenia gravis, a recent study compared 86 patients with the disease and 90 hospitalized controls, matched for…
Research
Pharmaceutical companies call for greater flexibility in assessing the toxicity of AAV-mediated gene therapy
On 06/03/2026
The question of the duration of clinical and biological monitoring after gene therapy remains under debate, particularly beyond 18 months: a survey was conducted among…
Research
BIND: a tool for better identifying and assessing central nervous system involvement in DMD
On 05/03/2026
An international consortium of researchers has developed and validated a new tool for studying disorders related to central nervous system damage (learning disorders, etc.) in…
Research
Limited efficacy of using AAV-U7 to deliver antisense oligonucleotides to the brains of DMD mouse models
On 05/03/2026
Researchers at the University of Versailles-Saint-Quentin tested antisense oligonucleotides (ASOs) targeting exon 51 skipping of the DMD gene in mdx52 mice, models of Duchenne muscular…
Research
Genotyping of a large cohort of neuromuscular patients in Iran
On 04/03/2026
Iranian clinicians and biologists report the results of genotyping 2,009 individuals with various neuromuscular diseases (including spastic paraplegia and hereditary ataxia): patients were recruited from…
Research
Organoids to understand the limited effectiveness of gene therapy in DMD
On 02/03/2026
Researchers at the Institute of Myology* and Genethon have developed muscle organoids, called ‘MYOrganoids’, that reproduce Duchenne muscular dystrophy (DMD) in order to study gene…
Research
Givinosat (Duvysat®) receives unfavourable opinion on reimbursement from the HAS in DMD
On 25/02/2026
Givinostat (Duvyzat®) has had conditional European marketing authorisation since June 2025 for boys aged 6 years and older with Duchenne muscular dystrophy (DMD) who are…
Research
SYNAPSE-CMT trial: NMD Pharma announces preliminary results
On 18/02/2026
The SYNAPSE-CMT Phase IIa clinical trial evaluated NMD670 (ignaseclant) administered orally twice daily, compared to placebo, in 81 adults with Charcot-Marie-Tooth disease (CMT1 and CMT2)…
Research
Lamotrigine appears to be effective over two years in channelopathies
On 16/02/2026
Used as an alternative to mexiletine in the treatment of non-dystrophic myotonia, lamotrigine shows lasting efficacy in a new British study: it involved 37 patients…
Research
Intrathecal gene therapy Itvisma effective in two Phase III trials in SMA
On 13/02/2026
In SMN1-related proximal spinal muscular atrophy (SMA), the intravenous treatment Zolgensma is indicated for infants and young children weighing less than 21 kg with type…
Events
The Institute acquires a new, latest-generation multimodal confocal microscope
On 12/02/2026
Bruno Cadot, researcher at the Center of Research in Myology and head of the MyoImage technology platform, oversaw the acquisition of a latest-generation Nikon AXR-NSPARC confocal…
Research
Effects of exoskeletons on mobility and motor function in MND patients
On 05/02/2026
Neuromuscular diseases (NMDs) cause progressive muscle weakness that significantly impairs patients’ functional abilities and quality of life. Romain Feigean, researchers in Neuromuscular Physiology and Evaluation…
Research
A new FDX2 mutation identified in a form of mitochondrial myopathy
On 05/02/2026
An Italian team studied the case of a 9-year-old patient with a mitochondrial myopathy called MEOAL (Mitochondrial Episodic Myopathy with or without Optic Atrophy and…
Research
French study identifies prognostic markers for interstitial lung disease associated with anti-synthase syndrome
On 04/02/2026
Conducted in seven reference or competence centres in north-eastern France, the TYPASS retrospective study included 132 adults with antisynthetase syndrome and interstitial lung disease: 39%…