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1385 news items
Research
Mapping dystrophin expression in the central nervous system
On 18/07/2025
In the context of the cognitive difficulties frequently observed in Duchenne muscular dystrophy (DMD), British researchers have reported work on the presence of dystrophin in…
Research
Towards European standardisation of registers and digital tools for myasthenia gravis
On 18/07/2025
European experts met in conclave at the European Neuromuscular Center (ENMC) to define standards for the identification and management of patients with autoimmune myasthenia gravis.…
Research
Proteomics as an alternative to muscle biopsy
On 17/07/2025
European researchers, including two from the Institute of Myology, report on progress in serum biomarkers for Duchenne muscular dystrophy (DMD): classical markers, such as creatine…
Research
EDG-5506 (sevasemten) stabilises functional scores in Becker’s myopathy
On 17/07/2025
EDG-5506 (Edgewise Therapeutics) is a small molecule which limits the recruitment of fast muscle fibres during muscular effort, protecting the muscle from possible mechanical damage.…
Research
Positive results in mice for a new gene therapy for type I SMA
On 16/07/2025
While Zolgensma® gene therapy in SMA is associated with a risk of cardiotoxicity and hepatotoxicity, another gene therapy EXG001-307 could represent a new, safer option…
Research
The risk of false positivity of RACh autoantibodies persists
On 15/07/2025
Italian biologists have looked retrospectively at the risk of obtaining ‘false positive’ autoantibody results when investigating patients suspected of having myasthenia gravis, the study was…
Research
The importance of in-depth genetic analysis in cases of congenital myopathies linked to the RYR1 gene
On 11/07/2025
On the basis of two paediatric cases of myopathies linked to the RYR1 gene and a review of the literature, a Japanese team has highlighted…
Research
Gene therapy on hold in Danon disease
On 10/07/2025
Despite encouraging results from a phase I trial of the RP-A501 gene therapy for cardiomyopathy in Danon disease, the phase II trial which had started…
Research
Myo-Guide: an online tool combining AI and MRI to help diagnose NMDs
On 10/07/2025
An international consortium has developed a web application based on artificial intelligence (machine learning) for the automated diagnosis of neuromuscular diseases using muscle MRI. It…
Research
A new series of cases of autosomal dominant calpainopathy
On 09/07/2025
French and Danish researchers have published data on patients suffering from primary calpainopathy and deviating from the usual pattern of inheritance: the 4 new cases…
Research
The use of an exoskeleton maximises the effect of nusinersen
On 07/07/2025
Japanese researchers report the results of a study comparing the outcome of 12 patients with SMA who had or had not undergone motor rehabilitation using…
Research
DMD: Translarna® loses its conditional authorisation in Europe and is no longer available in France
On 01/07/2025
On 28 March 2025, the European Commission announced the withdrawal of the European marketing authorisation for Translarna® in Duchenne muscular dystrophy. This concerned DMD patients…
Research
GenoTher Summit 2025: Shaping the Future of Gene Therapy together
On 30/06/2025
GenoTher Summit 2025, the first symposium organised by the GenoTher biocluster, was held on 11 June 2025 at the Génocentre in Évry-Courcouronnes. It brought together…
Research
Givinostat is approved for marketing in Europe for certain patients with DMD
On 30/06/2025
Givinostat, a histone deacetylase (HDAC) inhibitor that impacts muscle fibrosis and inflammation in Duchenne muscular dystrophy, is now conditionally approved in Europe under the name…
Research
New developments in the treatment of certain refractory inflammatory myositis
On 27/06/2025
German clinicians report a case of antisynthetase syndrome (ASyS) treated with several biotherapies: the patient, aged 45, had ASyS with signs of joint, muscle (myositis)…
Research
Towards a better understanding of the side effects associated with the use of CAR-T cells
On 26/06/2025
German clinicians have studied adverse side effects in patients with autoimmune diseases who have received CAR-T cells targeting the CD19 antigen: 39 adult patients, including…
Research
Tolerance of efgartimod in myasthenia gravis
On 25/06/2025
Chinese researchers have studied the adverse reactions observed when efgartigimod, a new-generation immunosuppressant used, among other indications, in refractory autoimmune myasthenia, is administered: their work…
Research
Is corticosteroid therapy effective on cardiac function in DMD? Review
On 24/06/2025
Chinese researchers wanted to know whether the long-term corticosteroid therapy used in Duchenne muscular dystrophy (DMD) could improve cardiac function: their work is based on…
Research
An optimised version of gene therapy being studied in SMA
On 23/06/2025
Beijing researchers report the results of preclinical studies on a new-generation gene therapy for SMA: the new transgene (AAV9-coSMN1) contains an optimised codon, carried by…
Research
Clinical presentations of immune-mediated necrotizing myopathy need to be better recognised
On 23/06/2025
American clinicians, including Andrew Mammen, the researcher behind the first descriptions of immune-mediated necrotizing myopathy (IMNM), take stock of this pathology and highlight its atypical…