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2414 news items
Research
Givinosat (Duvysat®) receives unfavourable opinion on reimbursement from the HAS in DMD
On 25/02/2026
Givinostat (Duvyzat®) has had conditional European marketing authorisation since June 2025 for boys aged 6 years and older with Duchenne muscular dystrophy (DMD) who are…
Events
ERN EURO-NMD webinar, 26 Feb.: Prof. Jim Bonham (UK)
On 20/02/2026
Genetics Month episode 4: Newborn Screening and Impact on Rare Diseases Thursday 26 February 2026 – 16:00-17:00 Paris time Prof. Jim Bonham (President of the…
Press releases
Klaudia Kuranda joins the Institute of Myology and Genethon to create a center of expertise dedicated to immunology
On 19/02/2026
An internationally recognized expert in the field of immunology and the development of gene therapies, Klaudia Kuranda is joining the AFM-Téléthon laboratories to establish and lead a new Immunology Center that will bring together…
Research
SYNAPSE-CMT trial: NMD Pharma announces preliminary results
On 18/02/2026
The SYNAPSE-CMT Phase IIa clinical trial evaluated NMD670 (ignaseclant) administered orally twice daily, compared to placebo, in 81 adults with Charcot-Marie-Tooth disease (CMT1 and CMT2)…
Events
First Network-Wide Event ENTRY-DM: a European launch in Paris to train a new generation of researchers
On 16/02/2026
The ENTRY-DM doctoral network (Interdisciplinary doctoral training on oligonucleotide-based therapies for myotonic dystrophy) officially launched in March 2025, and the 14 international doctoral students recruited…
Research
Lamotrigine appears to be effective over two years in channelopathies
On 16/02/2026
Used as an alternative to mexiletine in the treatment of non-dystrophic myotonia, lamotrigine shows lasting efficacy in a new British study: it involved 37 patients…
Events
ERN EURO-NMD webinar, 19 Feb.: Dr. Marcella Ner (Italy)
On 13/02/2026
Genetics Month episode 3: Genetics of Amyotrophic Lateral Sclerosis and Impact on New Therapies Thursday 19 February 2026 – 16:00-17:00 Paris time Dr. Marcella Neri…
Research
Intrathecal gene therapy Itvisma effective in two Phase III trials in SMA
On 13/02/2026
In SMN1-related proximal spinal muscular atrophy (SMA), the intravenous treatment Zolgensma is indicated for infants and young children weighing less than 21 kg with type…
Events
Full speed ahead to the 4th Muscle Week and the 2026 Muscle Conference!
On 12/02/2026
The Winter Olympic Games offer a timely reminder of a fundamental truth: muscle is not only responsible for athletic prowess and sporting performance, it is, above all, a major public health issue. By interacting with many organs,…
Events
The Institute acquires a new, latest-generation multimodal confocal microscope
On 12/02/2026
Bruno Cadot, researcher at the Center of Research in Myology and head of the MyoImage technology platform, oversaw the acquisition of a latest-generation Nikon AXR-NSPARC confocal…
Events
ERN EURO-NMD webinar, 12 Feb.: Dr. Fernanda Fortunato (Italy)
On 09/02/2026
Genetics Month episode 2: CPMS Use and Value for Genetic Case Discussions Thursday, 12 February 2026 – 16:00-17:00 Paris time Dr. Fernanda Fortunato (University Hospital…
Research
Effects of exoskeletons on mobility and motor function in MND patients
On 05/02/2026
Neuromuscular diseases (NMDs) cause progressive muscle weakness that significantly impairs patients’ functional abilities and quality of life. Romain Feigean, researchers in Neuromuscular Physiology and Evaluation…
Research
A new FDX2 mutation identified in a form of mitochondrial myopathy
On 05/02/2026
An Italian team studied the case of a 9-year-old patient with a mitochondrial myopathy called MEOAL (Mitochondrial Episodic Myopathy with or without Optic Atrophy and…
Research
French study identifies prognostic markers for interstitial lung disease associated with anti-synthase syndrome
On 04/02/2026
Conducted in seven reference or competence centres in north-eastern France, the TYPASS retrospective study included 132 adults with antisynthetase syndrome and interstitial lung disease: 39%…
Research
Positive results from the Phase III SAPPHIRE trial with apitegromab in SMA
On 02/02/2026
Apitegromab is a human monoclonal antibody that selectively inhibits myostatin activation to improve muscle function. Its efficacy was evaluated at one year in the Phase…
Research
Feedback on inflammatory myositis in the African population of Martinique
On 30/01/2026
Clinicians in Martinique report on their experience in the field of inflammatory myopathies occurring in the Caribbean population of African origin: the records of 174…
Research
Evrysdi, reimbursed for presymptomatic SMA
On 29/01/2026
According to the publication in the Official Journal of 13 January 2026, the conditions for reimbursement of Evrysdi (Risdiplam) in oral solution form have been…
Research
Is rituximab useful in ocular myasthenia gravis?
On 29/01/2026
Chinese clinicians report encouraging results regarding the use of rituximab, an anti-CD20 monoclonal antibody, in forms of ocular myasthenia gravis: their pilot study involved 9…
Events
“Michel Fardeau, a life under the microscope”: testimony from a pioneer in myology
On 28/01/2026
The French Society of Myology (SFM) has published « Michel Fardeau, a life under the microscope », a beautiful book (in French) dedicated to the founding father…
Research
An unprecedented paediatric case of myopathy linked to the HMGCR gene
On 28/01/2026
HMGCR is an enzyme involved in the metabolism of cholesterol and other metabolites. Until now, it was best known in connection with autoimmune necrotising myopathy…