Blog Archives
M&M’s – Muscle Monday Seminar – 1 Feb – Prof. Marco Narici (Italy)
Early biomarkers of muscle atrophy and of neuromuscular maladaptation during short-term inactivity in humans Monday 1st February 2021 – 12:00-13:00 Prof. Marco Narici (Dept of Biomedical Sciences, University of Padova, Italy) Hosted by Jean-Yves Hogrel On prior registration for people outside the Institute of Myology: medecine-umrs974-myologie@sorbonne-universite.fr > Further details on the presentation of Marco Narici
Ana Ferreiro is the winner of the Friedrich Wilhelm Bessel Prize
Ana Ferreiro received the prestigious Friedrich Wilhelm Bessel Research Prize awarded by the Alexander von Humboldt Foundation in Germany. Neurologist by training and holder of a thesis in molecular biology, Ana Ferreiro is head of the Fundamental and Translational Myology team at the Functional and Adaptive Biology Laboratory (BFA) of University of Paris / CNRS … [Read more]
MFM-32, MyoGrip, MyoPinch et ActiMyo, evaluation tools developed at the Institute of Myology sufficiently sensitive to detect a significant decline over 1 or 2 years in type II et III SMA
Spinal muscular atrophy (SMA) is a degenerative motor neuron disease which results in paralysis of very varying age and severity. There are four types depending on the age of onset and the best motor function achieved. Type I SMA is the most severe form, characterized by a very rapid decline in muscle strength. Type II … [Read more]
A composite score to assess the evolution of SMA in adults
SMA results in a table of paralysis of varying age onset and severity. It affects all ages including adults. As innovative treatments for SMA are launched, including for adults, the need for sensitive evaluation tools to assess small changes over short periods of time, such as in slowly evolving type III and IV AMS, is … [Read more]
Intravenous injection of AAV-microdystrophin SRP-9001 induces dystrophin production in muscles but does not statistically demonstrate the functional benefits observed at one year
SRP-9001 (rAAVrh74.MHCK7.micro-dystrophin) developed by the Sarepta Therapeutics laboratory were announced in a press release dated January 7, 2021. This double-blind, three-part trial evaluates the efficacy, tolerability and safety of one dose of SRP-9001 administered by infusion in 41 boys with DMD, aged 4 to 7 years, placebo controlled. In Part 1 now completed, 20 children … [Read more]
Classical pharmacological clinical trials and the potential of gene therapy in ALS
Amyotrophic lateral sclerosis (ALS) is a critical and incurable disease that affects the upper and lower motor neurons. Despite remarkable progress in understanding the pathological mechanisms of the disease, classical pharmacological clinical trials have failed to effectively cure ALS over the last twenty years. Recently, two different gene therapy approaches have been approved for SMA … [Read more]
The Institute of Myology is recruiting a Radiographer (M/F)
Situé à Paris au cœur du plus grand centre hospitalier européen, la Pitié-Salpêtrière, l’Institut de Myologie est né en 1996 sous l’impulsion d’une association de malades et parents de malades, l’AFM- Téléthon. Son objectif : favoriser l’existence, la reconnaissance et l’essor de la myologie en tant que discipline clinique et scientifique à part entière. L’Institut … [Read more]
The most frequent abnormalities of the SMN1 gene would concern exons 3 or 6 of the SMN1 gene in Brazil
Data from the literature have established the cause of SMA: 95% of people have a homozygous loss of SMN1 (no copy of the SMN1 gene) and 5% a heterozygous loss of SMN1 (absence of the SMN1 gene on one of the chromosomes and mutation on the other). A retrospective study was carried out in Brazil on … [Read more]
M&M’s – Muscle Monday Seminar – 25 jan – Mario Amendola, PhD (France)
Ex vivo editing of human hematopoietic stem cells for erythroid expression of therapeutic proteins Monday 25 January 2021 – 12:00-13:00 Mario Amendola, PhD (Therapeutic genome editing’ group,UMRS951, Evry, France) Hosted by : Maria-Grazia Biferi On prior registration for people outside the Institute of Myology: medecine-umrs974-myologie@sorbonne-universite.fr > Further details on the seminar of Mario … [Read more]
Validation of a new approach for the non-invasive assessment of muscle volume – Interview with D. Bachasson and J.-Y. Hogrel
The Institute of Myology teams have just published, in the Journal of Cachexia, Sarcopenia and Muscle, the results of a study demonstrating the significant potential of a non-invasive method to measure muscle volume. Interview with Damien Bachasson and Jean-Yves Hogrel, of the Institute of Myology’s Neuromuscular Physiology and Evaluation Laboratory. What is the overall context of … [Read more]
