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1385 news items
Research
Ventilatory weaning and myasthenic crisis: the experience of Raymond Poincaré University Hospital
On 22/10/2024
Clinicians in the western Ile-de-France region have retrospectively analyzed the clinical and paraclinical data of 126 myasthenia gravis patients who presented with a first myasthenic…
Research
Study of a Russian cohort adds to the phenotypic description of MATR3-linked distal myopathy
On 22/10/2024
Matrin-3 deficiency can give rise to a form of amyotrophic lateral sclerosis (ALS) or distal myopathy with vocal cord paralysis (VCPDM). Russian clinicians report the…
Research
Common in South India, GNE myopathy is associated with a founder effect
On 21/10/2024
Clinicians in southern India, working with other international research teams, have compiled clinical, biological and genetic data from a very large cohort of adult patients…
Research
Granulomatous myositis: a rare form of inflammatory myopathy
On 21/10/2024
French researchers report the clinical and histological data and immunological profile of 26 patients diagnosed with granulomatous myositis: the age of onset was generally very…
Research
L-carnitine and creatine show therapeutic potential in animal models of laminopathy
On 18/10/2024
Amino acid derivatives have been shown to be beneficial in a number of myopathies, including collagenopathies and congenital myopathies. Taiwanese researchers have explored the therapeutic…
Research
A new mouse model of Becker muscular dystrophy
On 18/10/2024
Canadian researchers have developed a new mouse model partially deleted for the gene encoding dystrophin (DMD gene) with the aim of imitating Becker muscular dystrophy:…
Research
Comparative data on vamorolone and prednisone for adrenal suppression
On 16/10/2024
Long-term corticosteroid therapy remains the reference treatment for Duchenne muscular dystrophy (DMD), but is associated with numerous side-effects, including resting of the hypothalamo adrenal axis.…
Research
New data on FOP flare-ups and the efficacy of garetosmab
On 16/10/2024
According to a post-hoc analysis of the results of the Lumina-1 phase II trial of garetosmab versus placebo in fibrodysplasia ossificans progressiva (FOP) : almost…
Research
Subcutaneous route non-inferior to intravenous for efgartgimod in myasthenia gravis
On 15/10/2024
In the Adapt-SC trial, 110 adults with generalised myasthenia gravis received a weekly subcutaneous (SC) or intravenous (IV) injection of efgartgimod (Vyvgart®) for 4 weeks.…
Research
Encouraging results from a pilot study of thiamine in CMT
On 14/10/2024
Thiamine, or vitamin B1, is important for nerve cell function. Administered as a dietary supplement, it is thought to have beneficial effects in a number…
Research
Salbutamol is effective in mouse models of collagenopathy
On 14/10/2024
Comme montré lors de recherches précédentes dans la SMA, le salbutamol est un candidat potentiel pour un repositionnement médicamenteux dans les maladies neuromusculaires avec des…
Research
Myostatin in SMA: few variations after treatment with nusinersen
On 14/10/2024
Researchers have studied the levels of endogenous circulating myostatin in patients with SMA, at a time when several anti-myostatin drugs are being tested: myostatin and…
Research
A tool for assessing swallowing disorders in SMA
On 11/10/2024
Italian clinicians report their experience in the endoscopic exploration of swallowing disorders in patients with type 1 proximal spinal muscular atrophy (SMA) who have not…
Research
Mixed results of gene therapy in two older children with SMA type 1
On 10/10/2024
British clinicians report their real-life experience of two children who received onasemnogene replacement gene therapy abeparvovec (OA) at a later age than usual: these two…
Research
A new ‘European’ form of oculopharyngodistal myopathy linked to the ABCD3 gene
On 10/10/2024
A study has identified 8 unrelated families, including 35 members suffering from a new form of oculopharyngodistal myopathy (OPMD) linked to the ABCD3 gene. This…
Research
Spinal cord organoids developed for SMA
On 08/10/2024
In recent years, the development of organoids, small 3D structures that resemble the whole organ, has enabled us to understand how they develop and function.…
Research
Can efgartigimod also be used for myasthenic attacks?
On 07/10/2024
Three publications published during the summer of 2024 report the use of efgartigimod (Vyvgart®), a neonatal anti-Fc receptor (FcRn), to treat acute decompensations of autoimmune…
Events
The Institute’s researchers and clinicians attend the 29th WMS Congress
On 04/10/2024
The 29th International Congress of the World Muscle Society (WMS) will be held from 8 to 12 October 2024 in Prague, Czech Republic. The Institute…
Research
Analysis of a large French cohort of patients with mitochondrial diseases
On 04/10/2024
The French network of diagnostic laboratories for mitochondrial diseases (MitoDiag) has published the results of a clinical and genetic study (panel, WGS and/or WES on…
Research
The results of the evaluation of omigapil in congenital muscular dystrophies have been published
On 03/10/2024
CALLISTO was a phase I trial designed to assess the safety, tolerability and pharmacokinetics of omigapil for 12 weeks in 20 children with congenital muscular…