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1670 news items
Research
A plea for neonatal screening for DMD
On 06/10/2025
In a comprehensive article, American specialists analyse practices and prospects for newborn screening for Duchenne muscular dystrophy in the United States: this screening is technically…
Research
Improvement in motor function with Evrysdi® in presymptomatic SMA
On 03/10/2025
Approved for spinal muscular atrophy (SMA), the efficacy of Evrysdi® in presymptomatic forms remained uncertain. In the international RAINBOWFISH open-label trial, 26 genetically diagnosed infants…
Research
Long-term IV immunoglobulins may reduce the incidence of cancer
On 02/10/2025
According to an analysis of follow-up data from 436 patients with autoimmune myasthenia gravis (MG) and 102 with chronic inflammatory demyelinating polyradiculoneuritis (CIDP), receiving intravenous…
Research
The SMA France Registry for collecting real-life or long-term data
On 01/10/2025
As of 22 July 2024, the SMA France Registry had collected data from 1,259 people with spinal muscular atrophy (SMA) through 59 participating reference centres,…
Research
Immunomodulatory mesenchymal cells useful in myasthenia gravis
On 01/10/2025
A group of researchers coordinated by the Institute of Myology has focused on a small contingent of cells and their potential use in the treatment…
Research
Sustainable cardioprotection with utrophin in DMD
On 30/09/2025
Scientists have evaluated a gene therapy based on micro-utrophin, a protein similar to dystrophin, which is deficient in Duchenne muscular dystrophy (DMD). This therapy was…
Events
LGMD Awareness Day: 30 September
On 29/09/2025
The 11th Limb Girdle Muscular Dystrophy Awareness Day takes place on 30 September. Organised by the LGMD Awareness Foundation, the aim of this day is…
Research
Mitochondrial abnormalities, a possible marker of unfavourable progression in inflammatory myopathies
On 29/09/2025
The presence of mitochondrial abnormalities is now a criterion for the diagnosis of sporadic inclusion myositis. It is also a predictive marker of progression in…
Research
Two new treatment approaches for progressive ossifying fibrodysplasia
On 24/09/2025
An ultra-rare disease, fibrodysplasia ossificans progressiva (FOP) results from gain-of-function mutations in the ACVR1 or ALK2 gene, which lead to the formation of heterotopic ossifications.…
Research
Genetic factors may influence the risk of developing myasthenia gravis, as well as its expression
On 23/09/2025
A retrospective study of the records of 281 patients with autoimmune myasthenia gravis followed by an Israeli specialist centre between 2000 and 2022 reveals that:…
Research
Unfavorable opinion on marketing authorisation and suspension of clinical trials in Europe for Elevidys in DMD
On 22/09/2025
Over the summer, the European Medicines Agency (EMA) issued an unfavourable opinion on the conditional marketing authorisation of Elevidys, a microdystrophin gene therapy for ambulatory…
Events
M&M’s – Muscle Monday Seminar – 22 Sept. – Dr Nicolas Wein (France)
On 22/09/2025
AAV.U7snRNA as a platform to treat neuromuscular disorders 22 September – from 12 to 1pm (Paris time) Dr Nicolas WEIN (Nantes, France) More information on…
Research
The benefits of physical activity are confirmed in neuromuscular patients
On 19/09/2025
In the Netherlands, researchers enrolled 44 inactive adults with various neuromuscular diseases (muscular dystrophy,congenital myopathy, etc.) in a six–month home training programme combining aerobic exercise…
Research
Launch of the MitoGether website, a reference for genetic mitochondrial diseases
On 17/09/2025
To celebrate the World Mitochondrial Disease Week (September 15th to 21st), the MitoGether consortium, comprising 12 patient associations and their families including AFM-Téléthon, is launching…
Research
Tadalafil and sildenafil have beneficial effects in patients with primary mitochondrial diseases
On 17/09/2025
Following the identification of a patient with Kearns-Sayre syndrome who reported significant clinical improvements while taking tadalafil, a Hungarian-American research team studied the efficacy of…
Events
World Myositis Day: 21 September
On 16/09/2025
Every year since 2001, 21 September has been World Myositis Day. Myositis is an autoimmune muscle disease that can affect adults and children. It causes…
Research
An inflammatory factor in Charcot-Marie-Tooth disease
On 16/09/2025
Two French publications suggest that an inflammatory mechanism may be involved in certain forms of Charcot-Marie-Tooth disease (CMT), which are generally considered to be exclusively…
Events
International Myotonic Dystrophy Awareness Day
On 15/09/2025
International Myotonic Dystrophy Awareness Day takes place every year on 15 September. It aims to raise awareness on this neuromuscular disease among the medical profession,…
Research
Review of current and emerging therapeutic strategies in DMD
On 15/09/2025
Duchenne muscular dystrophy (DMD) is a serious, progressive genetic disorder. It is caused by mutations in the DMD gene that result in the absence of…
Research
Researchers from the Institute attending the ESGCT conference
On 15/09/2025
The 32nd Annual Meeting of the European Society for Gene & Cell Therapy (ESGCT) will be held in Seville, Spain, from 7 to 10 October…