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1670 news items
Patient care
Increasing efficacy of Elevidys in DMD
On 10/03/2026
L’essai de phase III EMBARK avait présenté des résultats non significatifs contre placebo pour le critère principal à un an, ce qui avait valu un avis défavorable de l’Agence…
Research
An increased risk of urinary disorders in cases of autoimmune myasthenia
On 10/03/2026
In order to study immune disorders in autoimmune myasthenia gravis, a recent study compared 86 patients with the disease and 90 hospitalized controls, matched for…
Patient care
Enhancing diagnosis delivery in NMDs: insights from patient experiences
On 09/03/2026
The disclosure of a diagnosis is a pivotal event in a patient’s care pathway and has been the subject of extensive research across various medical…
Events
A football legends’ match in benefit of ALS research
On 09/03/2026
The Institut de Myologie Association is partnering with AS SAINT-ÉTIENNE, the organiser of the Match of the Heroes, which will take place on 31 May…
Events
ERN EURO-NMD webinar, 12 March: Prof. Martin Turner (UK)
On 09/03/2026
EXPERTS-ALS – a Bayesian biomarker-led drug screening platform in ALS Thursday 12 March 2026 – 16:00-17:00 Paris time Prof. Martin Turner (University of Oxford, UK) >…
Patient care
A pregnancy documented in SMA type I
On 09/03/2026
A retrospective study describes eight pregnancies in six women with severe SMA, who were all wheelchair-dependent, including one case of type I SMA. The three…
Patient care
Neurodevelopmental disorders in children with treated SMA type I: a point of attention
On 06/03/2026
Around twenty international experts on SMA gathered at a workshop organised by the European Neuromuscular Centre (ENMC) to review the neurodevelopmental disorders observed in a…
Research
Pharmaceutical companies call for greater flexibility in assessing the toxicity of AAV-mediated gene therapy
On 06/03/2026
The question of the duration of clinical and biological monitoring after gene therapy remains under debate, particularly beyond 18 months: a survey was conducted among…
Events
ERN EURO-NMD webinar, 5 March: Prof. Ammar Al-Chalabi (UK)
On 05/03/2026
Autonomic and non-motor symptoms in ALS Thursday 05 March 2026 – 16:00-17:00 Paris time Prof. Ammar Al-Chalabi (King’s College London, UK) > + infos Organized by…
Research
BIND: a tool for better identifying and assessing central nervous system involvement in DMD
On 05/03/2026
An international consortium of researchers has developed and validated a new tool for studying disorders related to central nervous system damage (learning disorders, etc.) in…
Research
Limited efficacy of using AAV-U7 to deliver antisense oligonucleotides to the brains of DMD mouse models
On 05/03/2026
Researchers at the University of Versailles-Saint-Quentin tested antisense oligonucleotides (ASOs) targeting exon 51 skipping of the DMD gene in mdx52 mice, models of Duchenne muscular…
Patient care
German-speaking real-world data on the use of innovative therapies in SMA
On 04/03/2026
Researchers consulted the SMArtCARE registry, which focuses on SMA in Germany, Austria and Switzerland, in the context of innovative therapies available since 2017: data from…
Research
Genotyping of a large cohort of neuromuscular patients in Iran
On 04/03/2026
Iranian clinicians and biologists report the results of genotyping 2,009 individuals with various neuromuscular diseases (including spastic paraplegia and hereditary ataxia): patients were recruited from…
Patient care
A case of Lambert-Eaton syndrome successfully treated with efgartigimod
On 03/03/2026
Chinese clinicians report the case of a 73-year-old patient diagnosed with Lambert-Eaton myasthenic syndrome (LEMS) who underwent treatment with a latest-generation immunosuppressant: the patient met…
Research
Organoids to understand the limited effectiveness of gene therapy in DMD
On 02/03/2026
Researchers at the Institute of Myology* and Genethon have developed muscle organoids, called ‘MYOrganoids’, that reproduce Duchenne muscular dystrophy (DMD) in order to study gene…
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…
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…