Blog Archives
Results of phase II trial of nipocalimab in myasthenia gravis published
Following an initial presentation at the 2021 American Academy of Neurology meeting, the final (and more moderate) results of the Vivacity-MG trial were published in the journal Neurology in January 2024 : 57 of the 68 participants completed 57 days of treatment with placebo or nipocalimab 5, 30 or 60 mg; the incidence of side-effects … [Read more]
Myology 2024 opening lecture by Drew Weissman, co-winner of the 2023 Nobel Prize in Medicine, and Jeffrey Chamberlain
Myology 2024 in Paris, join us now! Book your place at a preferential rate until 19 Feb., after this date, registration will be possible at the normal rate. Network with experts and explore the latest advances in the field of myology. Don’t miss the opening lecture with Drew Weissman from the University of Pennsylvania, co-winner … [Read more]
Pure distal myopathy involving the TDP-43 protein
A team of researchers and clinicians from Lyon report two unrelated families from the Ain region with late-onset distal myopathy with no signs likely to suggest associated motor neurone damage: A total of eight adult patients were studied, the disease being transmitted in an autosomal dominant fashion, with variable penetrance, The same missense variant of … [Read more]
Engineering muscle for human transplant – Interview with Bruno Cadot
In August 2023, Bruno Cadot won a call for proposals organised by the German National Innovation Fund (SPRIN-D) to finance his innovative project to create a synthetic muscle, combining chemistry and biology. Now that proof of concept has been achieved in mice, the next step is to make the transition to human transplants. Interview with Bruno … [Read more]
M&M’s – Muscle Monday Seminar – 12 February – Juanma Fernández Costa (Spain)
Organs-on-chip for muscular dystrophies: advanced models for evaluation of potential therapies 12 Febuary 2024 – From 12 to 1 pm Juanma Fernández Costa (BIST, Spain) Hosted by Valérie Allamand More information on the presentation and the speaker On prior registration for people outside the Institute of Myology: medecine-umrs974-myologie@sorbonne-universite.fr M&M’s seminars are organised by the … [Read more]
DMD: altered iron metabolism in cardiomyocytes corrected by deferoxamine or pioglitazone
A Polish team has investigated the mechanism involved in Duchenne cardiomyopathy by studying the transcriptome and proteome of exon 50-deleted cardiomyocytes derived from human iPS cells. She found : a decrease in mitoNEET protein levels ; an increase in labile iron in the cytoplasm and mitochondria; a decrease in ferroportin ; an increase in ferritin … [Read more]
ASPIRO trial: muscle lesions regress less quickly and less completely than in dogs
Muscle biopsies were taken before and after gene therapy in 10 children with myotubular myopathy participating in the ASPIRO trial. At 24 weeks after treatment, organelle localization was greatly improved, with no increase in myofibril size in most cases. On biopsy at 48 weeks post-treatment, the increase in myofibril size became statistically significant. However, the … [Read more]
A Franco-British study of spinal muscular atrophy not linked to chromosome 5q
Clinicians and geneticists from the Filnemus network, including members of the Institute of Myology, and from London pooled data from patients presenting with a picture compatible with spinal muscular atrophy, but where an SMN1 gene anomaly had been formally excluded: 71 patients from 65 families were included in the study, phenotypes were divided between proximal, … [Read more]
Biphosphonates and DMD: a review of the literature confirms the 2018 management recommendations
A review of the literature on the use of biphosphonates found in 19 publications concerning 1,010 children and adults with Duchenne muscular dystrophy (DMD) treated with corticosteroids: a high level of evidence that biphosphonates improve bone density in the areolar bone of the vertebral bodies of these patients; greater improvements in patients treated with intravenous … [Read more]
Gala Hope – Dance for Research
On 6 February, the Casino de Paris hosted the second Hope Gala. An exceptional evening that brought together the great names of French and international dance, in aid of research at the Institut de Myologie. Proceeds from the Gala will be donated to the Institut de Myologie Association. Initiated by Laura Arend, dancer and choreographer, … [Read more]
