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1699 news items
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.…
Events
Three prizes for the Institute at the WMS 2024 Congress
On 15/10/2024
Three speakers from the Institute of Myology were honored at the 29th International Congress of the World Muscle Society (WMS), held October 8-12, 2024 in…
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
Research
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…
Overview of the main myopathies that can begin in the over-50s
On 04/10/2024
A French review has set out to provide an overview of the most emblematic late-onset myopathies (LOMs), those which may appear after the age of…
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…
Research
ERDERA, a European partnership ushering in a new era in rare disease research – Interview with Dr Gisèle Bonne
On 02/10/2024
The European Research Alliance for Rare Diseases (ERDERA) is a European partnership that aims to improve the lives of millions of rare disease patients in…
Events
AcadeMYO 9-11 December 2024 online
On 26/09/2024
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…