Blog Archives
M&M’s – Muscle Monday Seminar – 15 Feb – WhiteLab Genomics
Data intelligence for cell & gene therapies development Monday 15 February 2021 – 12:00-13:00 WhiteLab Genomics (Company specialized in the development of an Artificial Intelligence platform dedicated to the field of genomic medicine, France) Hosted by Marc Bitoun On prior registration for people outside the Institute of Myology: medecine-umrs974-myologie@sorbonne-universite.fr > Further details on the seminar … [Read more]
A nucleoside-based treatment to improve respiratory impairment in TK2-deficient myopathy ?
Thymidin kinase 2-deficient myopathy (TK2-deficient myopathy) is a rare mitochondrial disease of varying severity. However, it causes very frequently a weakness of the diaphragm, the reason why half of patients have the first medical consultation, and has a poor prognosis. A nucleoside-based treatment allowed functional improvement in preclinical and compassionate use programme in a small … [Read more]
M&M’s – Muscle Monday Seminar – 8 Feb – Prof. Antonio Musarò (Italy)
Muscle homeostasis and regeneration: From cellular and molecular mechanisms to therapeutic opportunities Monday 8 February 2021 – 12:00-13:00 Prof. Antonio Musarò (Medical and Biotechnology School, Sapienza University of Rome, Italy) Hosted by Sestina Falcone On prior registration for people outside the Institute of Myology: medecine-umrs974-myologie@sorbonne-universite.fr > Further details on the seminar of Antonio Musarò
Release of the 83rd newsletter from the Institute
2021: life, let’s put some muscle into it, together Dear patients and families, Dear donors, Dear partners, We wish you a very happy New Year and excellent health. We hope this New Year brings you the energy for renewal needed to accomplish the projects close to your heart, and we hope these … [Read more]
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]
