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1385 news items
Clinical activity
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…
Clinical activity
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…
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…
Clinical activity
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…
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…
Research
Encouraging results for efgartigimod in juvenile myasthenia gravis
On 03/06/2026
Approved in France since 2022, efgartigimod (Vyvgart®) is currently indicated for adults with generalised autoimmune myasthenia gravis with anti-RACh antibodies. A multicentre retrospective study conducted…
Research
Researchers from the CRM’s REDs team attended the IDMC-15 conference
On 02/06/2026
The 15th International Myotonic Dystrophy Consortium Congress (IDMC-15), which brings together international scientists specialising in myotonic dystrophy every two years, was held this year from…
Research
For a comprehensive overview of diseases associated with the GBE1 gene
On 01/06/2026
American clinicians, in collaboration with the patient organisation APBD Research Foundation, have investigated muscle-affecting disorders linked to the GBE1 gene, which encodes the glycogen branching…
Research
In some forms of myositis, a decline in vital capacity during the first year is a poor prognostic sign
On 29/05/2026
American rheumatologists have investigated myositis complicated by interstitial lung disease, focusing in particular on the predictive value of respiratory function measurements over time: a cohort…
Research
CAR-T cells are effective in reducing autoantibodies in myasthenia gravis
On 29/05/2026
German researchers have developed and tested CAR-T cells using serum from patients with myasthenia gravis: this novel cell therapy aimed to enable effector cells to…
Research
A comparative analysis of antibody assays in a large Brazilian cohort of myositis patients
On 28/05/2026
Rheumatologists reviewed the clinical and immunological data of 351 adults diagnosed with inflammatory myopathy, with the aim of determining the prevalence of antinuclear antibodies by…
Research
Tools to better understand the deterioration of walking in hereditary or acquired neuropathies
On 27/05/2026
Researchers in Lille studied gait parameters in patients with Charcot-Tooth disease type 1A (33) and chronic inflammatory demyelinating polyneuropathy (CIDP) (31) under specific conditions: their…
Research
The MG-ADL scale is not the most appropriate tool for assessing respiratory impairment in myasthenia gravis
On 26/05/2026
Danish and French clinicians investigated the diagnostic and predictive value of the Myasthenia Gravis Activities of Daily Living (MG-ADL) scale for respiratory function in patients…
Research
A new case of adult myopathy associated with the ACTA1 gene
On 21/05/2026
French, Romanian and Russian researchers, including experts from the Institute of Myology, report the clinical, histological and genetic findings of an adult diagnosed with muscular…
Research
Autism spectrum disorders in children with SMA type 1 are not that rare
On 20/05/2026
Brazilian clinicians sought to determine the prevalence of autism spectrum disorders (ASD) among children with the most severe form of SMA type 1 who had…
Research
Significant variability in autoantibody tests
On 19/05/2026
Autoantibody testing is a key step in the diagnosis of many autoimmune diseases, including myositis. Against this backdrop, an international consortium of laboratories responsible for…