Blog Archives
DMD: Translarna® loses its conditional authorisation in Europe and is no longer available in France
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 aged 2 and over who were still walking and had a nonsense genetic anomaly in the DMD gene. In France, the decision was taken on 17 June 2025 by the … [Read more]
M&M’s – Muscle Monday Seminar – 7 July – Cedric Moro (France)
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 information on the presentation and the speaker On prior registration for people outside the Institute of Myology: medecine-umrs974-myologie@sorbonne-universite.fr M&M’s seminar organised by the Center of Research in Myology, at … [Read more]
GenoTher Summit 2025: Shaping the Future of Gene Therapy together
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 more than 350 key players in the gene therapy ecosystem for a day of scientific exchange and strategic reflection. More than 20 international experts from France, the United States, the … [Read more]
Givinostat is approved for marketing in Europe for certain patients with DMD
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 Duvysat™. It concerns: DMD patients, aged 6 and over, ambulant, on corticosteroid therapy; and requires further efficacy results to be produced on ambulant patients. In the EPIDYS trial, the group … [Read more]
New developments in the treatment of certain refractory inflammatory myositis
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) and respiratory (interstitial lung disease) disease, with the presence of autoantibodies directed against the Jo-1 antigen, an initial treatment with CAR-T cells directed against CD19 lymphocytes resulted in remission for … [Read more]
Towards a better understanding of the side effects associated with the use of CAR-T cells
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 six with inflammatory myopathy of autoimmune origin, were selected for this retrospective study. 30 of them developed a cutaneous and/or renal intolerance syndrome within 10 days of CAR-T cell injection. … [Read more]
A look back at the 26th AcadeMYO summer school
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 from 16 to 20 June. Every year, the Institute organises a 5-day summer school (AcadeMYO) to enable French and foreign professionals (18 countries* represented this year) to follow a condensed … [Read more]
Tolerance of efgartimod in myasthenia gravis
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 is based on pharmacovigilance data contained in the American database managed by the FDA (Food and Drug Administration), which is freely accessible and reports all types of adverse events linked … [Read more]
Is corticosteroid therapy effective on cardiac function in DMD? Review
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 a critical review of studies in the field published between 2000 and 2024, 21 studies were selected, 13 of which, corresponding to 1,814 DMD patients, argued in favour of a … [Read more]
M&M’s – Muscle Monday Seminar – 30 June – Kay Ohlendieck (Ireland)
Proteomic profiling of skeletal muscle aging Monday 30 June 2025 – 12h00-13h00 Pr Kay Ohlendieck (Maynooth University Faculty of Science & Engineering, Ireland) More information on the presentation and the speaker On prior registration for people outside the Institute of Myology: medecine-umrs974-myologie@sorbonne-universite.fr M&M’s seminar organised by the Center of Research in Myology, at the … [Read more]
