Support our Foundation of Myology project
1655 news items
Research
Research into biomarkers to differentiate Becker and Duchenne muscular dystrophies
On 01/09/2025
Swedish and Dutch researchers used mass spectrometry to try to identify proteomic profiles that would distinguish between the two most common types of dystrophinopathy, namely…
Research
The importance of measuring autoantibodies directed against rituximab in myasthenia gravis
On 29/08/2025
Neurologists in Nice report on their experience in the long-term treatment of autoimmune myasthenia gravis, particularly in the use of a monoclonal anti-CD20 antibody (rituximab…
Research
Reports of muscle lipidosis associated with sertraline and ranozaline
On 29/08/2025
American clinicians report their experience with two drugs, sertraline and ranozaline, which have caused cases of lipidosis-type myopathy: 10 cases of muscular lipidosis were recorded…
Research
First descriptions of cases of congenital myopathy linked to the dystonin gene
On 28/08/2025
An international consortium of researchers including experts from the Institute of Myology reports the identification of a new form of early-onset congenital myopathy linked to…
Research
Conditioned mesenchymal cells to alleviate Myasthenia Gravis
On 28/08/2025
Mesenchymal stromal cells (MSCs) are promising tools for the treatment of autoimmune and inflammatory diseases. These multipotent stromal cells possess innate immunomodulatory properties that can…
Research
NKG-001, a new generation gene therapy being tested in SMA
On 27/08/2025
NKG-001, a novel gene therapy product featuring a next-generation AAV vector (cceAAV for covalently closed-end double-stranded AAV), was tested intravenously in two children with proximal…
Research
A new international registry for patients with myasthenia gravis
On 27/08/2025
MGBase is a registry created in 2021 to compile online data on myasthenic patients worldwide: it is directly inspired by a registry developed for multiple…
Research
Few trinucleotide expansions are responsible for CMT disease in the UK
On 27/08/2025
English and Italian researchers have used high-throughput sequencing to examine unresolved cases of Charcot-Marie-Tooth disease (CMT): CGG expansions in the NOTCH2NLC and LRP12 genes have…
Research
Towards a broader phenotypic spectrum in type 0B muscular glycogen storage disease
On 26/08/2025
An international consortium of researchers reviews an ultra-rare form of muscular glycogen storage disease linked to the GYS1 gene encoding glycogen synthase: clinical and biological…
Research
A cardiac micro-pump useful in cases of decompensated cardiomyopathy in DMD
On 26/08/2025
American researchers report the successful implantation of a temporary Impella 5.5 endocavitary micro–pump developedby Abiomed: the patient was a 14–year–old boy with Duchenne muscular dystrophy,…
Research
Specific autoantibodies can predict response to treatment in dermatomyositis
On 25/08/2025
French researchers have identified specific autoantibodies associated with myositis that may predict the response to treatment of dermatomyositis with Janus kinase (JAK) inhibitors (JAKi): 39…
Research
Three new cases of the p.Ser85Cys mutation in the MATR3 gene in a form of distal myopathy
On 25/08/2025
French myologists report the case of three adults (two of whom are related) of Portuguese origin, who were treated in France for a slowly progressive…
Research
FOXK2: a new gene for congenital myopathy with ptosis
On 25/08/2025
Chinese researchers report for the first time mutations in the FOXK2 gene in five unrelated families: the clinical phenotype was that of an autosomal dominant…
Research
The Danish experience with myasthenia gravis
On 25/08/2025
Danish researchers report the results of a large epidemiological study on mortality observed in myasthenia gravis between 1985 and 2020: the information was drawn from…
Research
Gene therapy and DMD: a possible link with cardiac inflammation?
On 25/08/2025
Following the serious side effects observed during trials of micro–dystrophin gene therapy using an AAV viral vector in Duchenne muscular dystrophy (DMD), researchers at the…
Research
DMD: Canakinumab reduces certain blood markers but not IL1b
On 14/08/2025
Canakinumab (Ilaris®) is a monoclonal antibody that neutralises interleukin 1 beta (IL1β), an inflammatory marker that is highly expressed in Duchenne muscular dystrophy. It is…
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…