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1678 news items
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…
Events
A look back at JDF 2025, a meeting place for patients, families, experts and volunteers
On 09/07/2025
The annual Journées des Familles event organised by the AFM-Téléthon at the Parc Floral near Paris was held on 4 and 5 July. Nearly 1,700…
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…
Events
M&M’s – Muscle Monday Seminar – 7 July – Cedric Moro (France)
On 01/07/2025
Skeletal muscle as an endocrine organ during exercise Monday 7 July – 12h00-13h00 Cedric Moro, PhD (Institute of Metabolic and Cardiovascular Diseases, Toulouse, France) More…
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…
Events
A look back at the 26th AcadeMYO summer school
On 25/06/2025
The 26th edition of the Institute of Myology’s summer school has just come to an end. It was held face-to-face at the Institute of Myology…
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…
Events
M&M’s – Muscle Monday Seminar – 30 June – Kay Ohlendieck (Ireland)
On 24/06/2025
Proteomic profiling of skeletal muscle aging Monday 30 June 2025 – 12h00-13h00 Pr Kay Ohlendieck (Maynooth University Faculty of Science & Engineering, Ireland) More information…
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…
Events
Satish B. Moparthi, co-winner of the ‘Grandes Avancées Françaises en Biologie’ award from the French Academie des Sciences
On 20/06/2025
Satish Babu Moparthi, a post-doctoral fellow in the ‘Muscle cell organisation and therapy of centronuclear dominant myopathy’ team at the Center of Research in Myology…
Research
Long-term efficacy of nusinersen varies according to the frequency of injection
On 20/06/2025
Japanese researchers have compared two protocols, Japanese and European, for administering nusinersen in the treatment of SMA: for regulatory reasons, the Japanese protocol involves only…