Blog Archives
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]
SNOW-P: an Australian programme to manage eating behaviours in DMD
The SNOW-P programme, which stands for Supporting Nutrition and Optimising Wellbeing Programme, was co-constructed with carers of young boys with Duchenne muscular dystrophy. Fifty-three of them responded to a survey: 48% thought their son was overweight, which led to complexes (71%) and a negative impact on his self-esteem (64%) and mobility (57%); although healthy eating … [Read more]
DMD and rhabdomyosarcoma share similarities
Canadian researchers have written a book chapter summing up the knowledge acquired in the field of muscle stem cells (also known as satellite cells): these cells, located at the periphery of the muscle fibre, are the basis of muscle regeneration, they have been studied here in the two very different contexts of Duchenne muscular dystrophy … [Read more]
A remote study of the validity of the North Star functional scale
The COVID-19 pandemic provided an opportunity to rethink the methods used to assess functional scores in neuromuscular patients, particularly those with Duchenne muscular dystrophy (DMD): American researchers studied the validity of remote administration of the NSAA (North Star Ambulatory Assessment) scale in the form of a streaming video under the supervision of a physiotherapist, the … [Read more]
A look back at a week of action around muscle
From 1 to 7 June, a host of events were organised across France during Muscle Week to raise awareness of the importance of muscle for our health. This 2nd edition of the event received special recognition, as it was designated the Grande Cause Nationale 2024, dedicated to physical activity and sport (“Move 30 minutes a … [Read more]
