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1678 news items
Research
Myasthenia gravis in France seen through the national health data system prism
On 07/06/2024
In order to assess the incidence and prevalence of myasthenia gravis in France, a group of researchers from Auvergne analyzed data contained in the national…
Research
Effect of sirolimus on muscle in inclusion myositis observed by MRI and spectroscopy – Interview with Harmen Reyngoudt
On 06/06/2024
Harmen Reyngoudt is co-director of the Institute’s NMR imaging and spectroscopy laboratory. He has just published an article* in J Cachexia Sarcopenia Muscle on the…
Research
The wide variety of clathrin assemblies
On 06/06/2024
Clathrin is a protein that forms triskels that assemble into honeycomb-like networks on the plasma membrane, but also on internal membranes, such as the Golgi…
Research
CAR-T cell-based dual therapy successfully tested in refractory myasthenia gravis
On 05/06/2024
In a Letter to the Editor, Chinese clinicians report the case of a 64-year-old myasthenic patient who benefited from CAR-T cells targeting both the BCMA…
Research
The value of eculizumab in refractory myasthenia gravis and a possible switch to ravulizumab
On 04/06/2024
Japanese clinicians report their real-life experience of administering new anti-complementary molecules in a series of 36 adults with a refractory form of myasthenia gravis (3%…
Research
When the benefits of hot baths in DMD are revisited
On 03/06/2024
Brazilian physiotherapists have studied variations in physiological parameters in patients with Duchenne muscular dystrophy (DMD) in a seated position, in an aqueous environment or in…
Research
Inhibition of a cardiac microRNA improves fibrosis and function in a mouse model of DMD
On 31/05/2024
American and South Korean researchers have studied the myocardial dysfunction observed in Duchenne muscular dystrophy. Their work involved a transgenic mouse with a combined but…
Research
Preserving oral health when taking anti-CD20 drugs
On 30/05/2024
A team from Nice University Hospital reports: a series of six patients aged 34 to 79 who presented with oral disorders (pain, dental abscess or…
Research
Newborn screening for DMD as seen by various stakeholders
On 30/05/2024
Australian researchers wanted to know how newborn screening (NBS) for Duchenne muscular dystrophy (DMD) would be perceived at a time when innovative, supposedly effective therapies…
Events
Institute seminar – 31 May – Dr Lucy Collinson (UK)
On 29/05/2024
Volume electron microscopy and correlative imaging – how to find the needle in the haystack Friday 31 May 2024 – 11h00-12h00 – Institute of Myology, Paris…
Research
SMA with 4 copies of SMN2, a form with infantile onset?
On 29/05/2024
A study of data from 268 patients with 4 copies of the SMN2 gene from the SMArtCARE registry showed : an average age of first…
Research
Prevalence of epilepsy in dystrophinopathies revised downwards
On 28/05/2024
According to a retrospective study carried out in Spain on data from 416 patients with Duchenne muscular dystrophy, Becker muscular dystrophy or asymptomatic hyperCKemia :…
Research
Characterisation of Ku-positive myositis reveals a degree of heterogeneity
On 28/05/2024
An international consortium, including clinicians from the Institute of Myology, pooled clinical and biological data from patients suffering from a rare form of inflammatory myopathy,…
Research
Opto-acoustic imaging for spinal muscular atrophy
On 27/05/2024
German researchers have applied a technology designed to explore, non-invasively, patients suffering from proximal spinal muscular atrophy linked to the SMN1 gene (SMA) and benefiting…
Research
A Europe-wide analysis of Zolgensma® side effects
On 24/05/2024
Between January 2019 and September 2023, the pharmacovigilance database of the European Medicines Agency collected 661 individual reports of adverse reactions linked to Zolgensma®, representing…
Research
TRAPPC11 diseases reviewed
On 23/05/2024
On the occasion of the description of a Mexican case with an anomaly in the gene encoding the TRAPPC11 complex, a team from Mexico City…
Research
Cipaglucosidase alfa and miglustat in Pompe disease: two-year data
On 22/05/2024
Cipaglucosidase alfa, or Pombiliti®, is a recombinant enzyme being tested in Pompe disease in combination with miglustat (Opfolda®). The combination of the two drug candidates…
Events
ERN EURO-NMD webinar, Prof. Dr. Annemieke Aartsma-Rus (The Netherlands)
On 22/05/2024
Genetic therapies and therapy developments for muscular dystrophies (DMD, LGMD) Wednesday 29 May 2024 – 16:00 – 17:00 Paris time Prof. Dr. Annemieke Aartsma-Rus (Leiden…
Research
Results of a phase I gene therapy trial in giant axon neuropathy
On 21/05/2024
The dose escalation trial evaluated the effects of a single intrathecal injection of four escalating doses of scAAV9/JeT-GAN in 14 patients over the age of…
Research
The importance of developing strategies to manage altered facial expression in FSH
On 21/05/2024
Facial impairment is present in 75% of people with facioscapulohumeral muscular dystrophy (FSHD), and represents a significant psychosocial burden for a third. Given this distress,…