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1385 news items
Research
HAS opinion for Evrysdi® (risdiplam)
On 26/10/2021
The opinion of the Transparency Committee concerning the reimbursement of Evrysdi® (or risdiplam) has just been published. In SMA type I, II and III, Evrysdi®…
Research
ManNAc in GNE myopathy: new positive results
On 25/10/2021
A first phase I clinical trial of ManNAc (a precursor of sialic acid) in 22 people with GNE myopathy followed for 8 months, showed the…
Research
Reducing oxidative stress confines the development of symptoms in GDAP1-related CMT in mice
On 25/10/2021
The GDAP1 gene is involved in several forms of CMT (including CMT 2K and CMT 4A). It encodes a mitochondrial protein. After screening in vitro…
Research
Next-generation pacemaker successfully implanted in an adult with DMD
On 25/10/2021
Heart complications are part of the natural history of Duchenne muscular dystrophy (DMD), especially in adulthood, where they are responsible for a significant number of…
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…
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…
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
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…
Research
Study of the day-to-day life impact of autoimmune myasthenia gravis from the patient’s point of view
On 27/09/2021
Myasthenia gravis (MG) is a rare, chronic disease mediated by immunoglobulin G antibodies that causes crippling muscle weakness. As with most rare diseases, there is…