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1689 news items
Research
CAR-T cells have led to a remission lasting at least 19 months in three patients with myasthenia gravis
On 14/10/2026
The anti-CD19 CAR-T cells KYV-101 are being evaluated in a Phase II/III trial involving 66 patients with refractory generalised autoimmune myasthenia gravis. Initial results for…
Teaching
Registration is now open for the online Winter AcadeMYO (14 -16 Dec. 2026)
On 12/10/2026
Join us for this unique opportunity to perfect your skills in myology, an increasingly exciting discipline dedicated to neuromuscular science!All professionals manifesting some interest in…
Clinical activity
SMA: a new treatment targeting muscle has just been approved for marketing in the US
On 12/10/2026
Isembyld (apitegromab) has been granted US marketing authorisation for the treatment of SMA On 11 September 2026, the US Food and Drug Administration (FDA) granted…
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…
Clinical activity
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…
Clinical activity
Pompe disease: stabilisation of motor function two years after switching to avalglucosidase alfa
On 08/10/2026
A French study analysed real-world data from 47 adults with late-onset Pompe disease who switched to avalglucosidase alfa (Nexviadyme) after treatment failure with alglucosidase alfa…
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…
Clinical activity
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…
Clinical activity
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…
Clinical activity
Inflammatory myopathies: a new technology helping with diagnosis
On 06/10/2026
Plasma proteomics studies to assess the clinical manifestations and tissue damage associated with certain inflammatory myopathies An international consortium of researchers, including a French team,…
Clinical activity
Intermittent positive pressure ventilation 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
Myopathies inflammatoires : certaines affections auto-immnunes ont un risque non négligeable de complications pulmonaires
On 05/10/2026
Autoimmune conditions that test positive for anti-Ku antibodies carry a significant risk of pulmonary complications Clinicians from ten centres within the Assistance-Publique Hôpitaux de Paris (APHP) compiled and compared the clinical and laboratory data of 154 adults…
Clinical activity
What CPK levels are genuinely 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,…
Clinical activity
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,…
Clinical activity
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
Myasthenia gravis with anti-RACh+ antibodies: gefurulimab successfully completes Phase III trials
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…
Clinical activity
Steinert disease: initial results from the Phase III trial of del-desiran
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…
Clinical activity
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 progressive ossifying fibrodysplasia
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:…