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1699 news items
Documentation
Advances in congenital muscular dystrophies – 2025
On 12/08/2025
This document presents a selection of congenital muscular dystrophy research news from these last 12 months: ongoing observational studies and clinical trials, scientific and medical…
Research
The particularities of dermatomyositis on black skin
On 07/08/2025
The prevalence of dermatomyositis is higher in people with dark skin. After analysing 100 cases published since 1951, Canadian authors note that for this population:…
Documentation
Advances in Charcot-Marie-Tooth disease – 2025
On 04/08/2025
This document presents a selection of congenital muscular dystrophy research news from these last 12 months: ongoing observational studies and clinical trials, scientific and medical…
Research
Results of a trial of creatine monohydrate in a paediatric population with FSHD
On 04/08/2025
A randomised, double-blind, placebo-controlled trial was conducted in 13 children with facioscapulohumeral muscular dystrophy (FSHD) who received either creatine monohydrate or a placebo in a…
Research
Diseases related to BAG3: a common variant associated with a more severe form
On 31/07/2025
This is the largest study conducted in Europe on the initiative of clinicians at the Institute of Myology, involving 26 patients with mutations in the…
Research
The 2nd European Congress of CMT specialists will take place at the end of October in Belgium. Register now!
On 30/07/2025
Organised by the European Federation of CMT Associations, the University of Antwerp and the European CMT Research Association (ECRA), the second edition of this congress…
Research
DMD: launch of the pivotal phase of Généthon’s gene therapy trial
On 30/07/2025
The results of the gene therapy trial (GNT0004) conducted by Généthon for Duchenne muscular dystrophy were presented on 17 May at the 2025 conference of…
Research
An effective genomic editing approach in DMD
On 28/07/2025
In Duchenne muscular dystrophy (DMD), researchers used a CRISPR-Cas9 technique to correct the deletion of exon 52 of the DMD gene by targeting exon 53…
Research
Prevalence of GNE myopathy probably underestimated
On 25/07/2025
GNE myopathy (GNEM) remains a particularly rare neuromuscular disease (with an estimated 1 to 9 cases per 1 million), probably because it remains poorly understood…
Research
French experience of spinal surgery in patients with type II SMA
On 24/07/2025
Researchers at the Centre de Référence des maladies neuromusculaires at Trousseau Hospital in Paris have studied data from patients with SMN1 type II-related proximal spinal…
Events
Lecture: Experimental models in research, advances and ethical issues
On 24/07/2025
REGISTRATION Free access, in person, upon prior registration (no video conferencing) As part of its reflections on Science, ethics and innovation in the field…
Research
Genomic screening at birth: a revolution in the making
On 23/07/2025
Screening for rare diseases at birth using molecular biology is becoming a reality in several countries. The Screen4Care project brought together a group of experts…
Research
Antibodies to MuSK CRD are pathogenic in a mouse model of myasthenia gravis
On 23/07/2025
Myasthenia gravis (MG) is caused by autoantibodies directed mainly against the acetylcholine receptor (AChR) or the MuSK receptor tyrosine kinase located at the neuromuscular junction.…
Research
Inhibition of glutamate dehydrogenase as a new therapeutic approach in DMD
On 22/07/2025
European and Chinese researchers have focused on the glutamate pathway as a possible therapeutic target in Duchenne muscular dystrophy (DMD): this molecule plays an important…
Research
MoCo MRF T1-FF: a new approach to accurately assess upper body muscle tissues using NMR despite respiratory motion
On 21/07/2025
Over the last decade, MR Fingerprinting (MRF) has emerged as an effective paradigm for the rapid and simultaneous quantification of several parameters using MRI. This…
Research
Mapping dystrophin expression in the central nervous system
On 18/07/2025
In the context of the cognitive difficulties frequently observed in Duchenne muscular dystrophy (DMD), British researchers have reported work on the presence of dystrophin in…
Research
Towards European standardisation of registers and digital tools for myasthenia gravis
On 18/07/2025
European experts met in conclave at the European Neuromuscular Center (ENMC) to define standards for the identification and management of patients with autoimmune myasthenia gravis.…
Research
Proteomics as an alternative to muscle biopsy
On 17/07/2025
European researchers, including two from the Institute of Myology, report on progress in serum biomarkers for Duchenne muscular dystrophy (DMD): classical markers, such as creatine…
Research
EDG-5506 (sevasemten) stabilises functional scores in Becker’s myopathy
On 17/07/2025
EDG-5506 (Edgewise Therapeutics) is a small molecule which limits the recruitment of fast muscle fibres during muscular effort, protecting the muscle from possible mechanical damage.…
Research
Positive results in mice for a new gene therapy for type I SMA
On 16/07/2025
While Zolgensma® gene therapy in SMA is associated with a risk of cardiotoxicity and hepatotoxicity, another gene therapy EXG001-307 could represent a new, safer option…