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2414 news items
Research
DMD: first human study of DT-DEC01 cell therapy
On 04/12/2023
A Polish-American team working in collaboration with the laboratory Dystrogen Therapeutics has published the results of administering a new cell therapy product, DT-DEC01, to patients…
Events
M&M’s – Muscle Monday Seminar – 11 December – Fabio Pinna (Italy)
On 04/12/2023
NAD+ metabolism in cancer- and chemotherapy-induced cachexia 11 December 2023 – from 12 to 1pm Fabio Pinna (Department of Clinical and Biological Sciences, University of Torino,…
Research
DREAMS – a new Horizon Europe project brings hope to rare disease patients
On 29/11/2023
The objective of this 5-year project is to discover treatments for 5 rare neuromuscular disorders through a groundbreaking methodology combining Artificial intelligence (AI), stem cells…
Research
Garetosmab is effective in fibrodysplasia ossificans progressiva
On 27/11/2023
The Phase II LUMINA 1 trial assessed the safety and efficacy of garetosmab, a monoclonal antibody that blocks activin A signaling pathways, in 44 adults…
Research
Facial involvement is common in inclusion myositis
On 24/11/2023
Following on from previous work on facioscapulohumeral muscular dystrophy (FSHD), researchers in Marseilles have studied the characteristics of facial muscle deficit in people with inclusion…
Events
ERN EURO-NMD webinar, 30th Nov. – Dr. Montserrat Olivé Plana (Spain)
On 23/11/2023
Myopathology in protein aggregate myopathies and vacuolar myopathies 30th November 2023 – 4pm Paris time Dr. Montserrat Olivé Plana (Hospital Santa Creu i Sant Pau,…
Research
Testosterone in DMD improves the situation but does not normalise it
On 23/11/2023
The Newcastle team looked at the long-term follow-up of a cohort of 15 boys with Duchenne muscular dystrophy on corticosteroids who were treated with increasing…
Research
A new glycogen overload myopathy
On 22/11/2023
French researchers report the discovery of a gene responsible for a new myopathy beginning in childhood and accompanied by an accumulation of glycogen in the…
Research
The expertise of the NMR laboratory used to monitor the growth of a patient’s heart – Interview with Benjamin Marty
On 21/11/2023
Benjamin Marty is co-leader of the NMR and Spectroscopy Laboratory at the Institute’s Neuromuscular Investigation Center. Together with Yves Fromes, also a researcher in this…
Research
Mariko Okubo awarded at the WMS for the discovery of a potential new role for lamins – Interview
On 21/11/2023
Mariko Okubo is a post-doctoral researcher in the Myology Centre for Research in the « Myomatrix & Myonucleus Related Diseases: Genetics & Pathophysiology » team.…
Research
Biology of T cells in neuromuscular diseases: the case of DMD and ALS
On 21/11/2023
In neuromuscular diseases of different pathogenic origins, there is growing evidence of a close interaction between the immune system, the nerve and the muscle. The…
Research
IV immunoglobulins are particularly effective against the cutaneous signs of dermatomyositis
On 20/11/2023
The good results of the international ProDERM trial, evaluating Octagam® intravenous immunoglobulin versus placebo in 95 adults with dermatomyositis, led to an extension of the…
Research
A natural history of paediatric RYR1-related congenital myopathies
On 20/11/2023
Two major London paediatric neuromuscular centres carried out a retrospective study of 69 children with RYR1-related myopathy followed up between 1992 and 2019: 29 presented…
Research
No correlation between fatigue and severity in SMA?
On 20/11/2023
The results of an online survey on the impact of fatigue in proximal spinal muscular atrophy (SMA) carried out by the Cure SMA association among…
Research
Mixed efficacy results for SRP-9001 (Elevidys), a gene therapy approved in the USA for DMD
On 20/11/2023
In a press release dated 30 October 2023, Sarepta Therapeutics announced the initial efficacy results of the Embark trial, a phase III trial of Elevydis…
Research
Study identifies key criteria for optimum wheelchair adjustment
On 17/11/2023
A study financed by the AFM-Téléthon sought to identify the main positioning criteria guaranteeing the health and quality of life of adult wheelchair users with…
Research
Efgartigimod active on different muscle groups in myasthenia gravis
On 17/11/2023
A post-hoc analysis of the results of the Adapt trial, which was conducted in France in 167 adults with a generalised form of autoimmune myasthenia…
Research
The development of arimoclomol stops in inclusion myositis
On 17/11/2023
With a prevalence of 1 in 200,000 in Europe, sporadic inclusion myositis is the most common idiopathic myositis after the age of 50. It is…
Research
Louise Benarroch wins Elsevier Runner Up Award at WMS 2023 – Interview
On 16/11/2023
Louise Benarroch is a post-doctoral researcher in the “Myomatrix & Myonucleus Related Diseases: Genetics & Pathophysiology” team led by Gisèle Bonne in the Institute’s CRM.…
Press releases
Myotubular muscular dystrophy: trial results show efficacy on respiratory and motor function, but challenges remain
On 16/11/2023
Yesterday, The Lancet Neurology published the clinical results of a gene therapy trial conducted by Astellas Gene Therapies, using a drug candidate developed at Genethon,…