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2413 news items
Research
A better understanding of the barriers and enablers of physical activity in myasthenia gravis
On 19/08/2026
Led by Simone Birnbaum, a researcher at the Neuromuscular physiology and neuromuscular Evaluation Lab, in collaboration with the AFM-Telethon, the national MYaEX study involved 455…
Patient care
Significant global disparities in access to innovative treatments for SMA
On 23/07/2026
South African clinicians have compiled and analysed the difficulties in accessing innovative treatments for SMN1-related proximal spinal muscular atrophy in much of the non-Western world:…
Patient care
A combination therapy involving an antisense oligonucleotide and an HDAC6 inhibitor appears promising for SMA
On 21/07/2026
Researchers in Lyon have investigated, both in vitro and in vivo, the potential therapeutic synergy between the type 6 HDAC inhibitor (givinostat), which is already…
Patient care
TK2 deficiency: from the treatment of early-stage forms to the first benefits in late-stage forms
On 16/07/2026
On 31 March 2026, the European Commission granted a marketing authorisation (MA) under exceptional circumstances for Kygevvi (doxecitine and doxribtimine) for the treatment of mitochondrial…
Patient care
Towards a consensus-based integration of innovative therapies for myasthenia gravis?
On 13/07/2026
A group of European specialists in autoimmune myasthenia gravis set out to reach a consensus on the inclusion of new molecules (such as neonatal Fc…
Patient care
The benefits of the ketogenic diet in the treatment of myasthenia gravis
On 10/07/2026
German clinicians have investigated the benefits of the ketogenic diet (very high in fat and low in carbohydrates) in autoimmune myasthenia gravis, following on from…
Research
In myasthenia gravis, complement inhibitors increase life expectancy… and cardiovascular risk!
On 08/07/2026
Two German researchers investigated the benefits and potential complications – particularly cardiovascular ones – of a new class of drugs used to treat refractory forms…
Research
A review of myositis in the NEJM, authored by two French experts
On 24/06/2026
In line with their research over several decades and the advances they have already spearheaded, Professors Olivier Benveniste and Yves Allenbach (the Inflammatory Myopathies &…
Research
Results of the cemdisiran trial in myasthenia gravis have been published
On 22/06/2026
In 13 countries, including France, the Phase III Nimble trial evaluated cemdisiran, a small interfering RNA (siRNA) targeting complement component 5, administered subcutaneously, in patients…
Research
Increasing utrophin expression in DMD using CRISPR-Cas9
On 19/06/2026
One therapeutic approach for Duchenne muscular dystrophy (DMD) involves increasing the expression of utrophin to compensate for the absence of dystrophin, with which it shares…
Patient care
Recommendations for the management of neuropsychiatric disorders associated with DMD
On 16/06/2026
The management of neurodevelopmental and psychiatric disorders represents a major unmet need among patients with Duchenne muscular dystrophy (DMD). As part of the European BIND…
Research
An analysis of BNDMR data on myasthenia gravis
On 16/06/2026
A study of 3,963 patients with myasthenia gravis included in the National Rare Diseases Database (BNDMR) provides valuable insights into patient care at rare disease…
Patient care
Pioglitazone evaluated in inclusion body myositis during a phase I trial
On 15/06/2026
Pioglitazone, a selective agonist of the nuclear PPAR-γ receptors, was evaluated in a phase I trial involving 13 patients with inclusion body myositis, all of…
Events
M&Ms – Muscle Monday Seminar –15 June – Dr Cynthia Gagnon (Canada)
On 12/06/2026
Optimizing preparation for clinical trials in rare diseases through translational and interdisciplinary research: the NMD experience Monday 15 Juin 2026 from noon to 1pm (Paris…
Events
ERN EURO-NMD webinar, 18 June: Prof. Grazia Devigili (Italy)
On 12/06/2026
Episode 8 – Skin Biopsy in Small Fiber Neuropathy (in EURO-NMD Neuropathology Webinar Series) Thursday 18 June 2026 – 16:00-17:00 Paris time Speaker: Prof. Grazia Devigili…
Patient care
Very late-onset myasthenia gravis: a condition with a good prognosis that requires active treatment
On 11/06/2026
Very late-onset autoimmune myasthenia (VLOM) is becoming increasingly common: appears from the age of 65 onwards, is characterised by a male predominance and a rare…
Events
GenoTher Summit 2026, 17-18 June 2026, Paris
On 08/06/2026
On 17 and 18 June, the GenoTher biocluster is organising the GenoTher Summit 2026 – Accelerating the Future of Genetic Medicine in Paris. Bringing together…
Patient care
Risdiplam (Evrysdi) tablets: a new formulation now available in France
On 08/06/2026
The registration of risdiplam (Evrysdi) film-coated tablets in the French Official Journal has just been published. This new formulation will be available in pharmacies from…
Events
ERN EURO-NMD webinar, 11 June: Prof. Andoni Echaniz Laguna (France)
On 05/06/2026
Episode 7 – Amyloid Neuropathies and Therapeutic Approaches (in EURO-NMD Neuropathology Webinar Series) Thursday 11 June 2026 – 16:00-17:00 Paris time Speaker: Prof. Andoni Echaniz Laguna…
Research
Pyridostigmine and amifampridine evaluated versus placebo in myasthenia gravis
On 04/06/2026
Almost a century after Dr Mary Walker first used pyridostigmine to treat autoimmune myasthenia gravis: the randomised, randomised, double-blind IMPACT-MG trial in the Netherlands compared…