Blog Archives
Effect of sirolimus on muscle in inclusion myositis observed by MRI and spectroscopy – Interview with Harmen Reyngoudt
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 effect of sirolimus on muscle assessed by magnetic resonance imaging and spectroscopy. Interview with Harmen Reyngoudt. What is the background to this work? In order to make progress in clinical … [Read more]
The wide variety of clathrin assemblies
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 apparatus and tubular endosomes. Clathrin assemblies mainly regulate the intracellular trafficking of various protein components. Clathrin also has non-endocytic functions in cell adhesion through interactions with specific integrins. It contributes … [Read more]
CAR-T cell-based dual therapy successfully tested in refractory myasthenia gravis
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 and CD19 antigens: the patient had had a classic course of refractory myasthenia over the seven years since his diagnosis in 2016, a protocol of induced depletion of B lymphocytes … [Read more]
The value of eculizumab in refractory myasthenia gravis and a possible switch to ravulizumab
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% of a total cohort of 1,106 myasthenic patients): the 36 patients concerned had received eculizumab (Soliris®) as first-line treatment for an average of 35 months, clinical benefit was observed in … [Read more]
New techniques improve the diagnostic yield of high-throughput sequencing in neuromuscular diseases
Australian geneticists report on their experience in using more sophisticated high-throughput sequencing techniques (NGS for next-generation sequencing) or transcriptomic studies in the context of a molecular diagnostic laboratory for neuromuscular diseases: faced with the negativity of conventional whole exome sequencing (WES) or gene panel studies in certain patients, the authors used either RNASeq or whole … [Read more]
When the benefits of hot baths in DMD are revisited
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 an aerial environment: 16 DMD patients took part in an experiment in two different environments, measuring energy expenditure (in particular, oxygen consumption) and other vital parameters (functional capacity, heart rate), … [Read more]
Inhibition of a cardiac microRNA improves fibrosis and function in a mouse model of DMD
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 partial dystrophin and utrophin (mdx/utrn (+/-)) deficiency: an inhibitory microRNA was delivered to the animal via an AAV (adeno-associated virus) type 9 (AAV9) to counter the effects of miRNA25, an … [Read more]
Newborn screening for DMD as seen by various stakeholders
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 are being announced: 50 caregivers and 26 healthcare professionals took part in a survey, the vast majority (80%) said they were in favor of screening, even if few knew the … [Read more]
Preserving oral health when taking anti-CD20 drugs
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 fracture, periodontitis…) 10 days to two years after starting anti-CD20 therapy (rituximab or ocrelizumab) for autoimmune myasthenia, multiple sclerosis or neuromyelitis optica ; the Vigibase worldwide pharmacovigilance database has recorded … [Read more]
Institute seminar – 31 May – Dr Lucy Collinson (UK)
Volume electron microscopy and correlative imaging – how to find the needle in the haystack Friday 31 May 2024 – 11h00-12h00 – Institute of Myology, Paris Dr Lucy Collinson (Head of Electron Microscopy, Francis Crick Institute, London, UK) Auditorium of the Institut of Myology Babinski Building Hôpital La Pitié Salpêtrière Entrance from the 50/52 boulevard Vincent … [Read more]
