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1655 news items
Research
MRI signal abnormalities in adults with Brown-Vialetto-Van Laere syndrome
On 22/10/2021
Brown-Vialetto-Van Laere syndrome (BVVLS) is a very rare form of bulbspinal muscular atrophy which is treated with high doses of riboflavin. It results in motor…
Research
The Institute of Myology jointly discovers a new form of X-linked distal myopathy
On 20/10/2021
An international collaboration that included researchers from the Institute of Myology has discovered a new form of distal myopathy, in 10 men: with adult onset…
Research
Newborn screening for SMA becomes official in Belgium
On 20/10/2021
Several countries have already deployed pilot programs for newborn screening in the SMA, such as Germany, Australia, the United States, Taiwan … In Belgium, a…
Events
ERN EURO-RND webinar, October 21st: Gisele Bonne (France), Carles Hernandez-Ferrer (Spain), Katja Lohmann (Germany)
On 19/10/2021
Rare Diseases’ therapies integration in diagnostic support tools through the Treatabolome platform Thursday October 21st, 2021 – 4pm (Paris Time) Gisele Bonne (Sorbonne University –…
Research
The deterioration of respiratory and motor parameters is confirmed in non-walking patients with type III SMA
On 19/10/2021
SMA classically includes four types (I to IV) depending on the age of onset of the deficit and the maximum motor ability achieved by the…
Research
WMS 2021: fairly encouraging results of losmapimod in facio-scapulo-humeral myopathy
On 18/10/2021
A selective inhibitor of p38 α and β MAP kinases, losmapimod has been investigated in a phase II trial called ReDUX4, randomized, double-blind, placebo-controlled, lasting…
Research
Can physical exercise at home be useful in patients with myasthenia gravis?
On 18/10/2021
Myasthenia gravis is the result of an imbalance in the immune system, causing the production of autoantibodies that act against one of the building blocks…
Research
A Swedish study finds that MG treatment leaves unsatisfactory symptomatic state for patients in almost half of cases
On 18/10/2021
In Sweden, 1,077 patients with myasthenia gravis from the Genes and Environment in Myasthenia Gravis study (GEMG) cohort completed a 106-item self-administered questionnaire including the…
Research
Inflammatory myopathies: the molecular benefits of physical activity are revealed
On 16/10/2021
Appropriate physical activity is an integral part of the treatment of idiopathic myositis (or inflammatory myopathies), though the mechanisms of action being are not yet…
Research
WMS2021 and SMA: between innovative therapy and biotherapy
On 15/10/2021
New clinical trial data in SMA were discussed during the latest edition of the World Muscle Society (WMS) congress from September 20-24, 2021. Out of…
Events
ERN EURO-RND webinar, October 19th: Prof. Annemieke Aartsma-Rus (The Netherlands)
On 13/10/2021
Oligonucleotide therapies: a new class of drugs that allow precise genetic targeting Tuesday October 19th, 2021 – 3-4pm (Paris Time) Prof. Annemieke Aartsma-Rus (Department of…
Research
DMOP: a Dutch study specifies the evolutionary profile of the disease
On 11/10/2021
Oculopharyngeal muscular dystrophy (or OPDM) is a relatively rare myopathy transmitted on an autosomal dominant mode. It results in the gradual onset, as a rule…
Research
Gastrointestinal manifestations of Pompe disease: confirmation of smooth muscle involvement
On 11/10/2021
Pompe disease is an inherited disease linked to the dysfunction of a lysosomal enzyme involved in the physiological glycogen breakdown. Transmitted on an autosomal recessive…
Research
SMA type 1: preliminary results of FIREFISH, a trial evaluating risdiplam
On 07/10/2021
After one year of treatment with risdiplam (Evrysdi), the primary endpoint of the 2nd part of the FIREFISH trial was met. The improvements achieved last…
Research
Better known risks of Zolgensma
On 04/10/2021
Zolgensma® (onasemnogene abeparvovec) is the first gene therapy product to obtain marketing authorization in 2019 in a neuromuscular disease, namely SMA. Various serious side effects…
Research
Functionally repaired DMD heart cells with Crispr-Cas9
On 04/10/2021
A team of American researchers has treated lines of induced pluripotent stem cells taken from a patient with Duchenne muscular dystrophy (DMD) with a deletion…
Events
M&M’s – Muscle Monday Seminar – 11 October – Steve Wilton (Australia)
On 04/10/2021
Antisense oligomer induced spliceoform switching: from Duchenne muscular dystrophy to common serious inherited and acquired diseases Monday October 11th 2021 from 12am to 1pm. Steve Wilton…
Events
‘Fête de la Science’ – October 5-8 – ‘Les apéros au Labo’
On 01/10/2021
The operation, organized each year by the Ministry in charge of Education, Research and Innovation, aims to allow everyone to discover the world of science…
Events
Release of the 87th newsletter from the Institute – September 2021
On 30/09/2021
The 26th International Congress of the World Muscle Society, which brings together muscle experts from around the world, was held September 20-24, providing an opportunity…
Events
The Institute has acquired a state-of-the-art electron microscope
On 29/09/2021
Interview with Norma B. Romero and Stéphane Vassilopoulos Since July 2021, researchers, doctors and engineers/ technicians from the Institute of Myology have been learning to…