Blog Archives
Firdapse® in Myasthenia Gravis too?
3,4-diaminopyridine or amifampridine (Firdapse®) is already indicated in some myasthenic syndromes, congenital or autoimmune (Lambert-Eaton). In the past, case reports had alerted to the possible efficacy of this drug in myasthenia gravis as well. The results of an open-label clinical trial conducted in Italy in 15 adults with anti-acetylcholine receptor (AChR) myasthenia gravis support these … [Read more]
Barriers to heart transplantation in MELAS syndrome remain numerous
MELAS syndrome is associated with mitochondrial encephalomyopathy, elevated lactic acid and pseudo strokes. Impairment of cardiac and renal function is also frequently reported. Italian researchers retrospectively studied the cardiac status of 23 MELAS patients diagnosed in 1998 and 2021 : all had been referred to cardiology for evaluation of first hypertrophic and then dilated cardiomyopathy. … [Read more]
Apremilast, a useful additional treatment for refractory skin lesions in dermatomyositis
According to the results of a phase IIa trial conducted in the United States in 8 adult patients with dermatomyositis with well-treated resistant skin lesions and a Cutaneous Dermatomyositis Disease Area and Severity Index (CDASI) greater than 14 : the addition of 2 tablets per day of apremilast (Otezla®) 30 mg resulted in a reduction … [Read more]
Evidence for earlier prescription of rituximab in adult generalized myasthenia gravis
At a time when new therapeutic families are emerging in autoimmune myasthenia, an “old” treatment (rituximab) could play a new role. Indicated until now in refractory forms of the disease, it has just been evaluated in recent generalised myasthenia (12 months or less) by the phase III clinical trial Rinomax, conducted in Sweden in 47 … [Read more]
M&M’s – Muscle Monday Seminar – 21 November – Brian Glancy (USA)
Sustaining Power: Building Energy Networks in Striated Muscle Monday November 21st, 2022 – 14:00 – 15:00 Brian Glancy (Earl Stadtman Investigator – NHLBI (NIH National Heart, Lung, and Blood Institute) and NIAMS (National Institute of Arthritis and Musculoskeletal and Skin Diseases)) On prior registration for people outside the Institute of Myology:: medecine-umrs974-myologie@sorbonne-universite.fr More information on the … [Read more]
Evolution of autoantibodies in MG: a review of the literature
Myasthenia gravis (MG) is mostly related to the abnormal production of autoantibodies directed either against the acetylcholine receptor (ARch) or against the MuSK receptor, and much more rarely against other elements of the neuromuscular junction such as titin. A group of European experts, including two French representatives, one of whom is from the Institute of … [Read more]
The European Reference Network (ERN) EURO-NMD is looking for a Project Assistant (m/f)
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 diseases and conditions that require highly specialized treatment, as well as a concentration of knowledge and resources. Established in 2017 and co-funded by the European Commission, EURO-NMD is a European … [Read more]
Anemia and myasthenia gravis
Anemia and myasthenia gravis (MG) are two pathological situations more often reported in the female population. Their association is frequent and of multifactorial origin. Japanese clinicians and biologists sought to find out more by studying a cohort of patients who consulted the Keio University Hospital in Tokyo throughout 2021. 215 women diagnosed and/or followed for … [Read more]
Dystrophin restoration after AAV U7-mediated Dmd exon skipping is modulated by muscle exercise in a mouse model of severe DMD
A team of French researchers, involving researchers from the Institute, showed in a mouse model of severe DMD that voluntary exercise has an impact on a Dmd exon skipping approach and on muscle physiology. D2-mdx mice were administered an AAV-U7 snRNA intra-muscularly to correct the Dmd reading frame and then allowed to run in a … [Read more]
Symposium 2022 – The Gene Therapy Revolution Underway: The Latest Advances – Novembre 16th, 2022, Paris
The GTRU 2022, an international scientific symposium dedicated to the advances and challenges of gene therapy, will be held on Wednesday 16 November 2022 from 9 am to 6.30 pm at the Imagine Institute at Necker Hospital (Paris). This event called ” The Gene Therapy Revolution Underway : The Latest Advances ” is organised by … [Read more]
