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1678 news items
Research
CAR-T cells to treat severe forms of Lambert-Eaton syndrome
On 16/12/2024
German researchers report the case of a patient with Lambert-Eaton syndrome (or LEMS, a presynaptic disorder of the neuromuscular junction very often of paraneoplastic origin)…
Events
Report on the ‘1,000 researchers in schools’ operation
On 13/12/2024
The 12th edition of the ‘1,000 researchers in schools’ operation has just come to an end. This year it ran from 4 November to 6…
Research
New data on life expectancy in DM2
On 13/12/2024
Of the 125 Dutch patients with myotonic dystrophy type 2 (DM2) recorded in the Dutch neuromuscular database, 26 died between 2000 and 2023 : the…
Events
ERN EURO-NMD webinar, 19 Dec. : Prof. Edoardo Malfatti (France)
On 13/12/2024
Novel approaches and future developments in Neuromuscular Pathology Thursday 19 December 2024 – 16:00 – 17:00 Paris time Prof. Edoardo Malfatti (Paris-Est AP-HP, France) >…
Research
An algorithm to assist diagnostic for seronegative autoimmune myasthenia gravis
On 12/12/2024
At the 275th workshop of the European Neuromuscular Centre (ENMC), held in February 2024 in the Netherlands, experts and patient representatives met to review the…
Research
From diagnosis to treatment, antisynthetase syndrome is attracting international attention
On 11/12/2024
In France, the National Data Bank for Rare Diseases lists 1,156 patients followed up in Centres of Reference or Competence for an anti-synthetase syndrome (SAS),…
Research
Post-hoc news on efgartigimod in anti-RACh myasthenia gravis
On 10/12/2024
The phase III placebo-controlled Adapt trial and its open-label extension Adapt+ evaluated the efficacy of efgartigimod, an anti-FcRN, in generalised autoimmune myasthenia gravis. They ended…
Events
Tribute to Michel Fardeau, pioneer of myology
On 09/12/2024
Michel Fardeau passed away on 6 December at the age of 95. A medical researcher, he began his career as a Clinic Director at Paris…
Research
Methylation studies to help with the molecular diagnosis of FSHD
On 09/12/2024
The Italian consortium dedicated to facioscapulohumeral muscular dystrophy (FSHD) reports on its experience in integrating methylation studies into the routine diagnosis of this myopathy with…
Research
The case of premature twins with SMA who received early treatment
On 06/12/2024
American clinicians report their experience of gene therapy treatment of two twins with proximal spinal muscular atrophy (SMA) who were born prematurely. The twins were…
Research
DMD: French recommandations on corticoids
On 06/12/2024
On the basis of a review of the literature, the Filnemus neuromuscular rare diseases health network and the French Paediatric Neurology Society (SFNP), including clinicians…
Events
ERN EURO-NMD webinar, 12 Dec.: Prof. Dr. Norma Romero (France)
On 06/12/2024
The role of muscle biopsy in Congenital Myopathies Thursday 12 December 2024 – 16:00 – 17:00 Paris time Prof. Dr. Norma Romero (Institute of Myology,…
Research
Stamina analyses the treatment of myasthenia in ‘real life’ in France
On 05/12/2024
Based on data from the French National Health Data System (SNDS), the Stamina study reviewed the management of two groups of adults with autoimmune myasthenia…
Research
Three-dimensional movement analysis measures the effectiveness of gene therapy in SMA
On 04/12/2024
French clinicians report the development and results of a follow-up protocol for infants with SMA treated with gene therapy (onasemnogene abeparvovec or Zolgensma®): 23 children…
Events
ERN EURO-NMD webinar 5 Dec.: Prof. Werner Stenzel (Germany)
On 03/12/2024
The role of muscle biopsy in inflammatory myopathies Thursday 5 December 2024 – 16:00 – 17:00 Paris time Prof. Werner Stenzel (Charité – Universitätsmedizin, Berlin, Germany)…
Research
A new natural history study in SMA
On 03/12/2024
An international consortium of clinicians with expertise in SMA reports new natural history data in this disease: the study population consisted of 226 patients with…
Research
The UK experience of SMA gene therapy
On 02/12/2024
The UK SMA REACH consortium is reporting its experience in treating infants with SMA with abeparvovec onasemnogene (OA, Zolgensma®): 93 babies with type 1 SMA…
Events
Telethon 2024: 79,801,520 euros – the struggle goes on!
On 01/12/2024
The thirty hours of the 2024 Telethon came to a close with a total of 79,801,520 euros raised. A fantastic result for an event held…
Research
US recommendations for treating SMA
On 29/11/2024
At the initiative of Cure-SMA, the American association of patients suffering from SMN1-related proximal spinal muscular atrophy (SMA), experts have analysed all aspects of innovative…
Research
Neonatal screening of premature babies with SMA: the German experience
On 28/11/2024
German clinicians report on their experience in the management of premature newborns diagnosed during newborn screening for SMN1-related proximal spinal muscular atrophy (SMA): there are…