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1385 news items
Research
A new murine model of myasthenia gravis that is more effective and better suited to animal welfare
On 12/05/2025
The Myasthenia gravis, etiology, pathophysiology & therapeutical approaches team, led by Rozen Le Panse at the Institute of Myology’s Myology Research Centre, has developed a…
Research
Identification of effective, non-invasive MRI markers in LGMD R9
On 12/05/2025
French researchers from the Institute of Myology, Genethon and Atamyo, in collaboration with Dutch researchers, have studied several quantitative MRI parameters in order to identify…
Research
The benefit of reusing data from placebo groups in inclusion myositis
On 12/05/2025
American researchers associated with the Institute of Myology Research Centre have compiled and re-analysed the data accumulated in the placebo arms of several clinical trials…
Research
A revision of the classification of congenital myopathies in the light of recent discoveries
On 09/05/2025
The team at the Créteil Neuromuscular Disease Reference Centre takes stock of congenital myopathies in the light of the recent exponential increase in knowledge in…
Research
Immune-mediated necrotising myopathy in children, a Chinese picture
On 07/05/2025
A study conducted by a hospital in Beijing (China) has refined our knowledge of the phenotype of immune-mediated necrotising myopathy in paediatrics: 55 of the…
Research
Long-term data on the evolution of patients with congenital myopathy
On 05/05/2025
The Centre de référence des maladies neuromusculaires of the Institute of Myology conducted a single-centre observational study of 142 adult patients with congenital myopathy followed…
Research
Positive results with gene therapy in a monkey model of DMD
On 02/05/2025
A Chinese team has developed a rhesus monkey model of Duchenne muscular dystrophy, enabling it to evaluate a new gene therapy, with encouraging results. The…
Research
Overexpressing utrophin in DMD: a new therapeutic approach in the spotlight
On 30/04/2025
Chinese researchers have revived a therapeutic technique designed to over-express utrophin, an endogenous protein very similar to dystrophin: they used a genome-editing approach in several…
Research
Data from a cohort of 219 patients with distal myopathy provide further details on the characteristics of these diseases
On 30/04/2025
Spanish doctors have published the demographic, clinical, genetic and physiological data from the largest cohort of patients with distal myopathy. With 219 patients included, the…
Research
A significant advance in DMD cell therapy
On 29/04/2025
British researchers have succeeded in significantly improving the grafting of myogenic progenitor cells (MPC) into the muscle fibre of mouse models of Duchenne muscular dystrophy:…
Research
Towards a European register of autoimmune myasthenia gravis cases
On 29/04/2025
At a time when innovative therapies are being developed for refractory forms of autoimmune myasthenia gravis, the idea of setting up a European register is…
Research
Valproic acid improves exon skipping efficiency in DMD mouse models
On 28/04/2025
The major challenge of exon skipping therapy in Duchenne muscular dystrophy is to effectively deliver the antisense oligonucleotides to the targeted tissues, in this case…
Research
Repeated intrathecal injections of nusinersen and pain syndrome: can we anticipate its onset?
On 28/04/2025
South Korean researchers sought to identify predictive factors for the occurrence of a pain syndrome after long-term transforaminal injection of nusinernen in patients with SMA: 34…
Research
An English study on mortality in SMA in the era of innovative therapies
On 21/04/2025
The UK consortium dedicated to the care of young patients with SMA has carried out a retrospective mortality study using data from the national SMA…
Research
CMT X1: positive results in mice with a new gene therapy
On 14/04/2025
A Cypriot team has developed a new gene therapy called AAVrh10-hMPZ.GJB1 aimed at restoring connexin 32 expression in X-linked Charcot-Marie-Tooth disease (CMT X1) and tested…
Research
Axonal neuropathy with hereditary neuromyotonia linked to MPZ
On 07/04/2025
Until now, only recessive mutations in the HINT1 gene have been implicated in a form of axonal neuropathy with hereditary neuromyotonia. A Greek team reports…
Research
Idiopathic myositis: an increased and early risk of cardiovascular events in cases of cardiac involvement at diagnosis
On 04/04/2025
The Hôpital Saint-Antoine (Paris) conducted a retrospective study between 1997 and 2020 of 78 patients with idiopathic inflammatory myopathy (myositis). Of these, 42% had dermatomyositis,…
Research
A comparative study of costs between nusinersen and onasemnogene abeparvovec
On 04/04/2025
In association with the Novartis laboratory, which markets the onasemnogene abepavovec or OA (a gene therapy product prescribed for infantile spinal muscular atrophy linked to…
Research
Spinal cord epidural stimulation proves effective in type III SMA
On 04/04/2025
An initial study evaluated epidural stimulation of the spinal cord in three patients with type III spinal muscular atrophy (SMA). While motor deficits may persist…
Research
A candidate biomarker for mitochondrial diseases: serum chitotriosidase 1
On 03/04/2025
Elevation of serum chitotriosidase is a biomarker of lysosomal diseases, particularly in Niemann-Pick disease. Recently, the lysosomal component of mitochondrial diseases has been discussed. Serum…