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1686 news items
Research
The diagnostic and pathophysiological challenges of toxic vacuolar myopathies
On 09/10/2026
Clinician and researchers from the Institute of Myology, in collaboration with international researchers, have investigated myopathies induced by chloroquine (and its derivatives, referred to by…
Research
Muscle biopsy: a diagnostic technique that remains useful despite advances in genetics
On 08/10/2026
Italian researchers have examined the diagnostic value of muscle biopsy at a time when high-throughput sequencing (NGS) and immunoassays are increasingly relegating it to a…
Research
Stabilisation of motor function two years after switching to alfa-sialidase in Pompe disease
On 08/10/2026
A French study analysed real-world data from 47 adults with late-onset Pompe disease who switched to alfa-glucosidase (Nexviadyme) after treatment failure with alfa-glucosidase (Myozyme). Longer-term…
Research
Modulation of the muscle satellite cell pool (MuSC) by GDF5: a potential therapeutic benefit for DMD
On 08/10/2026
Researchers from the Center of Research in Myology* at the Institute have demonstrated the benefits of treatment with growth and differentiation factor 5 (GDF5) in…
Patient care
Digestive problems: not to be underestimated in Steinert disease
On 07/10/2026
A study assessed the frequency of gastrointestinal symptoms and their burden in 95 patients with the adult-onset form of Steinert disease. The authors recommend incorporating…
Patient care
MFM is now available as an app: pros and cons
On 07/10/2026
The teams in Lyon and Saint-Étienne, who designed and developed the Motor Function Measure (MFM), have announced that it is now available on a mobile…
Research
Plasma proteomics studies to assess the clinical manifestations and tissue damage associated with certain inflammatory myopathies
On 06/10/2026
An international consortium of researchers, including a French team, analysed the proteomic profiles of 201 patients seen in Stockholm who were suffering from inflammatory myopathy:…
Patient care
VPPI does indeed appear to have a positive effect on chest growth in children with type I SMA
On 06/10/2026
A French retrospective study assessed the effect of intermittent positive pressure ventilation (IPPV) using the Alpha 300® in seven children with type I spinal muscular…
Research
Autoimmune conditions that test positive for anti-Ku antibodies carry a significant risk of pulmonary complications
On 05/10/2026
Clinicians from ten centres within the Assistance-Publique Hôpitaux de Paris (APHP) compiled and compared the clinical and laboratory data of 154 adults with positive anti-Ku…
Research
At what levels are CPK levels actually considered pathological?
On 05/10/2026
British and Canadian researchers have sought to establish non-pathogenicity reference ranges for creatine phosphokinase (CPK) levels measured in routine clinical practice: According to the authors,…
Patient care
Incorporating pool-based exercises into the management of Steinert disease
On 02/10/2026
In a Canadian study, 22 adults with Steinert’s disease (myotonic dystrophy type 1 or MD1) were randomised into groups for either aquatic or land-based training,…
Patient care
Update to the national strategy for the genetic diagnosis of myopathies by Filnemus
On 01/10/2026
The French Rare Health Care for Neuromuscular Diseases Network (Filnemus) has published an update to its strategy for the genetic diagnosis of neuromuscular diseases, including…
Research
Gefurulimab successfully completes Phase III trials in myasthenia gravis with anti-RACh+ antibodies
On 01/10/2026
The Phase III PREVAIL trial evaluated gefurulimab (or ALXN1720) against a placebo in 20 countries, including France, in 249 adults with a generalised form of…
Research
Initial results from the Phase III trial of del-desiran in Steinert disease
On 29/09/2026
In September 2026, Novartis announced the initial results of the Phase III HARBOR trial evaluating delpacibart andedesiran (del-desiran or AOC 1001) in type 1 myotonic…
Patient care
Diabetes is more common in late-onset dermatomyositis, regardless of corticosteroid use
On 24/09/2026
A retrospective analysis was carried out using the medical records of 434 patients with late-onset dermatomyositis, matched with a control cohort. These results confirm the…
Research
An antisense strategy to specifically target one of the mutations responsible for FOP
On 23/09/2026
Canadian researchers have developed antisense oligonucleotides (DNA/RNA gapmers) designed to selectively target the R206H mutation in the ACVR1 gene – which is most commonly responsible…
Events
21 September marks World Myositis Day
On 21/09/2026
Today, 21 September 2026, marks the 25th International Myositis Awareness Day, organised by the Myositis Association 1 person in 7,000 has myositis worldwide Also known as ‘inflammatory myopathies’, myositis is linked to inflammation of an autoimmune origin:…
Events
M&M’s – Muscle Monday Seminar – 21 sept. – Dr Marzia Rossato (Italy)
On 15/09/2026
Sequencing Technologies: Opportunities from Whole-Genome to Targeted Analysis of Complex Genomic Variants and Epigenetic Modifications Monday 21 September 2026 – noon-1pm Dr Marzia Rossato (University of…
Research
Diaphragmatic ultrasound: a promising biomarker for the risk of respiratory complications in Steinert myotonic dystrophy
On 15/09/2026
French clinicians investigated diaphragmatic function in patients with Steinert’s myotonic dystrophy (DM1) using an ultrasound method, which is by definition non-invasive: The authors highlight the…