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1676 news items
Research
New results from the SUNFISH and JEWELFISH clinical trials with risdiplam in SMA
On 31/03/2023
In two publications released in February 2023, the results of two clinical trials evaluating risdiplam (Evrysdi) in SMA type II and III (SUNFISH trial) and…
Events
ERN EURO-NMD webinar, Apr. 6th – Prof. Werner Stenzel (Germany)
On 30/03/2023
Inflammatory Myopathies 6th April 2023 – 4pmParis Time Prof. Werner Stenzel (Charité – Universitätsmedizin Berlin, Germany) Registration: https://zoom.us/webinar/register/WN_sdHfvA2tSei3Nx_MvL-_qQ Organised by EURO-NMD in collaboration with ERN-RND
Research
Daratumumab, an anti-CD38 effective in two cases of severe refractory myositis
On 30/03/2023
The first case concerns a 27-year-old man in Canada with autoimmune necrotizing myopathy with anti-PRS: after 8 months of conventional treatment (glucocorticoids, methotraxate, IV immunoglobulins,…
Research
Results of a longitudinal study on the effectiveness of corticosteroid therapy in the non-ambulatory phase of DMD
On 30/03/2023
A longitudinal study published in December 2022 compared disease progression after the loss of walking in 86 boys with DMD with or without steroid therapy…
Research
The advantages of quantitative magnetic resonance imaging in Becker muscular dystrophy
On 30/03/2023
The natural history of Becker’s muscular dystrophy (BMD) is imperfectly known. In order to assess its evolution and with a view to therapeutic trials, researchers…
Research
Pyridostigmine tends to improve subjective fatigability, but shows no significant effect on muscle strength
On 29/03/2023
The SPACE clinical trial took place between 2016 and 2017 in the Netherlands. This single-centre, phase II, cross-over trial evaluated an 8-week course of pyridostigmine…
Research
Researchers from the Institute’s NMR laboratory at the SFRMBM 2023 conference
On 28/03/2023
The 6th conference of the French Society of Magnetic Resonance in Biology and Medicine (SFRMBM) was held in Paris from 27 to 29 March 2023.…
Research
Cross-sectional study of neuromuscular fatigue in autoimmune myasthenia – Interview with Simone Birnbaum
On 27/03/2023
Simone Birnbaum, a researcher at the Neuromuscular Physiology and Evaluation Laboratory directed by Jean-Yves Hogrel (NIC, Institute of Myology) has just published an article assessing neuromuscular fatigue…
Research
A new pathophysiological mechanism to explain asymmetry in mitochondrial myopathies
On 27/03/2023
Mitochondrial diseases are a heterogeneous group of disorders caused by alterations in mitochondrial DNA (missense mutations, sporadic large-scale deletions or mutations in nuclear maintenance genes).…
Research
Fiorella Grandi, winner of the prestigious Marie Sklodowska-Curie 2022 postdoctoral fellowship – Interview with F. Grandi and P. Smeriglio
On 24/03/2023
Fiorella Grandi started a postdoctoral fellowship in October 2022 in the Epigenetics and biotherapies for motor neuron disorders (ALS & SMA) group led by Piera…
Research
New long-term results of Zolgensma
On 24/03/2023
In a press release dated 20 March 2023, Novartis presents new results for Zolgensma (onasemnogene abeparvovec) from two 15-year follow-up studies: Study LT 001 is…
Research
Quantitative MRI to assess the evolution of muscle condition
On 22/03/2023
Clinical trials related to dystrophinopathies are increasingly using quantitative MRI to assess the evolution of muscle status in patients with these neuromuscular diseases. In this…
Events
The European Reference Network (ERN) EURO-NMD is looking for a Project Assistant (M/F)
On 16/03/2023
ERN EURO-NMD European Reference Networks (ERNs) are networks involving centers of expertise and health care providers across Europe. They aim to tackle complex or rare…
Events
5th Neuromuscular Translational Summer School – 10-14 July 2023 – Leiden, The Netherlands
On 14/03/2023
Organized by Euro-NMD and TREAT-NMD networks, this summer school offers lectures concerning the management of acquired and genetic neuromuscular diseases, the different steps of drug…
Events
A look back at the 27th SFNP meeting
On 13/03/2023
The 27th meeting of the Société Francophone du Nerf Périphérique (SFNP) took place on 10 & 11 March 2023 in Paris Bercy in the salons…
Research
The need for early recognition and treatment of primary carnitine deficiency
On 13/03/2023
Clinicians and biologists from Bordeaux report the observation of a four and a half month old child who presented a severe metabolic decompensation. The clinical…
Research
Artificial intelligence for the evaluation of movements in DMD
On 10/03/2023
Clinical trials using force or movement measurements are highly dependent on patient motivation. To overcome this bias, British researchers developed a biomedical device with whole-body…
Research
A new clinical trial on DM1 at the Institute of Myology
On 09/03/2023
The double-blind, placebo-controlled phase I/II ACHIEVE trial is evaluating DYNE-101. This drug candidate for Steinert’s disease is being developed by the company Dyne Therapeutics. It…
Events
M&M’s – Muscle Monday Seminar – 13 March – Eric T. Wang (USA)
On 09/03/2023
Repeat Expansions Instigate Supply Chain Issues in Nucleus and Cytoplasm 13th March 2023 – 12:00 – 13:00 Eric T. Wang (Dept of Molecular Genetics &…
Research
New drug causes immune-mediated necrotizing myopathy
On 09/03/2023
Immune-mediated necrotizing myopathy is of recent description and linked, in most cases, to exposure to cholesterol-lowering drugs such as statins. American clinicians report a novel…