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Research
An indirect comparison of two innovative compounds in the treatment of myasthenia gravis
On 21/06/2024
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…
Events
ERN EURO-NMD webinar, 27 June: Prof. Dr. Jan Kirschner (Germany)
On 20/06/2024
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,…
Research
Belgian recommendations for the management of myasthenia gravis
On 20/06/2024
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…
Events
M&M’s – Muscle Monday Seminar – 24 June – Kay Ohlendieck (Ireland)
On 19/06/2024
Proteomic tissue and biofluid markers of dystrophinopathy Monday 24 June – 12am – Institute of Myology, Paris Pr Kay Ohlendieck (Maynooth University, Ireland) More information…
Research
Successful use of CAR-T cells in simultaneous myasthenia gravis and Lambert-Eaton syndrome
On 19/06/2024
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…
Research
DMD and rhabdomyosarcoma share similarities
On 19/06/2024
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…
Research
SNOW-P: an Australian programme to manage eating behaviours in DMD
On 18/06/2024
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…
Research
A remote study of the validity of the North Star functional scale
On 18/06/2024
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):…
Research
Omega3s appear to improve the FOXP3 regeneration biomarker in DMD
On 17/06/2024
Supplementation with long-chain omega-3 polyunsaturated fatty acids (ω-3 LCPUFA) was tested in a double-blind, placebo-controlled study for six months in 31 boys with Duchenne muscular…
Research
Myasthenia gravis: mycophenolate and methotrexate better tolerated than azathioprine
On 17/06/2024
A UK national survey of 235 people with myasthenia gravis, 166 on azathioprine, 102 on mycophenolate and 40 on methotrexate, showed that : the most…
Research
An early marker of myocardial damage in DMD
On 17/06/2024
Catalan clinicians have studied the possibility of early detection of myocardial alterations in Duchenne muscular dystrophy (DMD): 23 adolescents with dystrophinopathy (14 DMD, 8 Becker,…
Research
Expert consensus on the assessment and management of adverse reactions to delandistrogene moxeparvovec (Elevidys®)
On 14/06/2024
Given the rapid and recent development of microdystrophin gene therapy treatments for Duchenne muscular dystrophy (DMD), there is little data on the side effects of…
Research
ENMC workshop rethinks diagnosis and clinical trial design in inclusion myositis
On 14/06/2024
Ten years after the publication of the diagnostic criteria for inclusion myositis, an international group of around thirty experts (including two from France) took part…
Research
Lessons from the long-term follow-up of over 200 adults with congenital myasthenic syndrome in France
On 13/06/2024
A retrospective study based on data from 235 adults collected by a total of 23 French expert centres, during a follow-up period averaging 34 years,…
Research
An update on good practice in molecular biology for FSHD
On 12/06/2024
Despite substantial advances in its pathophysiology, muscular dystrophy or facioscapulohumeral myopathy (FSHD) remains a complex muscular disease with at least two genes involved. An international…
Research
Results of losmapimod and antioxidants in FSHD
On 11/06/2024
New trial results have recently been published for facioscapulohumeral muscular dystrophy (FHSD). For losmapimod, they show that : in a phase II trial involving 80…
Research
Inclusion myositis and sirolimus: the final results of the Rapami trial pave the way for a phase III
On 10/06/2024
Sporadic inclusion myositis combines inflammatory processes and degeneration. It is the most common myositis after the age of 50, but also the only one that…
Research
Myasthenia gravis in France seen through the national health data system prism
On 07/06/2024
In order to assess the incidence and prevalence of myasthenia gravis in France, a group of researchers from Auvergne analyzed data contained in the national…
Research
Effect of sirolimus on muscle in inclusion myositis observed by MRI and spectroscopy – Interview with Harmen Reyngoudt
On 06/06/2024
Harmen Reyngoudt is co-director of the Institute’s NMR imaging and spectroscopy laboratory. He has just published an article* in J Cachexia Sarcopenia Muscle on the…
Research
The wide variety of clathrin assemblies
On 06/06/2024
Clathrin is a protein that forms triskels that assemble into honeycomb-like networks on the plasma membrane, but also on internal membranes, such as the Golgi…