Blog Archives
Improved pneumococcal vaccination coverage in inflammatory diseases in the United States
Faced with very low vaccination coverage rates, particularly against pneumococcus, in patients with inflammatory pathologies, American clinicians set up an action plan: the target population was patients with lupus or connective tissue diseases (including inflammatory myopathies such as dermatomyositis), whether or not they were being treated with immunosuppressive drugs, two types of vaccine (PCV13 and … [Read more]
Normal strength is not essential for a functional hand – Interview with Valérie Decostre
Valérie Decostre is a physiotherapist and researcher at the Neuromuscular Physiology and Evaluation Laboratory, directed by Jean-Yves Hogrel, at the Neuromuscular Investigation Center of the Institute of Myology. She has just published an article (1) examining the link between strength and muscle function of the hand in Duchenne muscular dystrophy (DMD) and spinal muscular atrophy … [Read more]
Cardiac myosin inhibitor improves skeletal muscle contractility in Laing myopathy
Mavacamten is a small molecule inhibitor of myosin ATPase activity, used in hypertrophic cardiomyopathies, which targets myocardial hypercontractility by reducing the number of myosin heads in a disordered relaxed configuration in favor of a predominantly super-relaxed state as in normal muscle. Mouse models carrying the MYH7 mutation most common in distal Laing myopathy display muscle … [Read more]
Nusinersen does not prevent hip instability in SMA type II
Researchers in Hong Kong investigated the extent to which treatment with intrathecal nusinersen influenced the risk of bilateral hip dislocation, a complication very frequently observed in spinal muscular atrophy type II (SMA II) linked to the SMN1 gene : 10 children with SMA type II were included in the study, which ran for three and … [Read more]
Gait in DMD now easier to analyze in real life
Californian researchers have developed an original, easy-to-use method for measuring gait parameters in young patients with Duchenne muscular dystrophy (DMD): a gyroscope contained in a commercial smartphone and a specific application were used to measure the person’s movements in the three planes of space, 15 young DMD patients (aged 3 to 16) and 15 healthy … [Read more]
An indirect comparison of two innovative compounds in the treatment of myasthenia gravis
Efgartigimod (Vyvgart®) and ravulizumab (Ultomiris®) are two new-generation monoclonal antibodies, anti-FcRN and anti-C5 respectively, designed to treat refractory forms of generalised autoimmune myasthenia with positive autoantibodies against the acetylcholine receptor. German clinicians have attempted to compare their efficacy and safety indirectly: the results of the two main studies of one (ADAPT trial) and the other … [Read more]
ERN EURO-NMD webinar, 27 June: Prof. Dr. Jan Kirschner (Germany)
Genetic therapies and therapy developments for SMA Thursady 27 June 2024, 16:00 – 17:00 Paris time Prof. Dr. Jan Kirschner (Medical Center – University of Freiburg, Germany) Gene Therapy webinar series Organised by EURO-NMD in collaboration with ERN-RND.
Belgian recommendations for the management of myasthenia gravis
Following the sharing of experience in the field, and in view of the very rapid development of innovative therapies, Belgian clinicians have drawn up national recommendations for the management of patients suffering from myasthenia gravis : these recommendations were drawn up in February 2024 and cover both generalised myasthenia and ocular myasthenia, they take into … [Read more]
M&M’s – Muscle Monday Seminar – 24 June – Kay Ohlendieck (Ireland)
Proteomic tissue and biofluid markers of dystrophinopathy Monday 24 June – 12am – Institute of Myology, Paris Pr Kay Ohlendieck (Maynooth University, Ireland) More information on the presentation and the speaker In person only: Amphithéâtre F 105 boulevard de l’Hopital 75013 Paris M&M’s seminar organised by the Center of Research in Myology, at the … [Read more]
Successful use of CAR-T cells in simultaneous myasthenia gravis and Lambert-Eaton syndrome
A German team has reported the treatment with autologous anti-CD19 CAR-T cells of two women with both autoimmune myasthenia and Lambert-Eaton syndrome who had failed to respond to multiple immunotherapies. The two patients, who were wheelchair-dependent before the transplant, regained full mobility, enabling them to cycle and go hiking in the mountains, in particular, within … [Read more]
