Blog Archives
New tools for assessing the follow-up of two forms of muscular dystrophy
Monitoring, either as part of the usual follow-up of patients with muscular dystrophy or as part of clinical trials, is largely based on the use of functional scores: researchers report, in association with Duchenne muscular dystrophy (DMD) patient associations, the development of an upper limb function scale centred on the needs and feelings of patients … [Read more]
Significant drug iatrogenicity in late myasthenia gravis
A retrospective study of the medical data of 493 people suffering from autoimmune myasthenia followed by the university hospital centres of Strasbourg, Lyon, Grenoble and Angers reveals that the disease began after the age of 70 for 28% of them. Among these older patients : 76% improved with treatment during follow-up, despite lower doses, 41% … [Read more]
Results of the phase II-III trial of AMO-02 in congenital DM1
The results of the international REACH-CDM trial involving 56 children aged between 6 and 16 with the congenital form of myotonic dystrophy type 1 were announced in a press release on 6 September 2023. The primary endpoint, the Clinician-Completed Congenital DM1 Rating Scale, was not met. A significant reduction in CPK enzymes was measured in … [Read more]
Right heart involvement in DMD is a poor prognostic factor
A study of the respiratory and systolic functions of the right ventricle on Doppler echo in 90 young men with Duchenne muscular dystrophy (DMD), with a median age of 27.5 years and a median vital capacity of 10% of theoretical, showed : right ventricular systolic impairment in 46 (51%); the presence of left ventricular damage … [Read more]
Senolytic molecules improve the behaviour of muscle stem cells in DM1
In DM1, muscle stem cells adopt the characteristics of senescent cells, including the secretion of senescence-associated secretory phenotype (SASP). A Canadian team showed that : serum levels of certain SASPs (including interleukin 6) are correlated with muscle weakness and functional limitations. administration of a senolytic product inhibiting BCL-XL leads to the death of senescent myoblasts … [Read more]
Infantile myasthenia remains ocular and usually evolves favourably
A retrospective study of 859 patients followed by a Shanghai hospital for Myasthenia gravis diagnosed before the age of 14 found : an ocular form in 97.8% of cases, which only generalized in 14% of patients; 21.7% were in stable complete remission, 15.3% in pharmaceutical remission, and 42% of patients had achieved minimal manifestation status … [Read more]
Tofacitinib effective on dermatomyositis skin lesions, but not really on muscle
A Canadian team conducted a single-center study of 41 people with refractory myositis treated with tofacitinib after failure of four to five immunosuppressants: 23 had classical dermatomyositis, 12 an amyopathic form and 6 polymyositis; tofacitinib produced statistically and clinically significant skin improvement in the dermatomyositis group, but had no significant effect on muscle in all … [Read more]
MYOLOGY 2024: registration and submission of abstracts now open
The website for the 8th edition of the international congress dedicated to neuromuscular science MYOLOGY 2024, to be held in Paris from 22 to 25 April 2024, is now open. Abstracts can be submitted on the website until 30 November 2023. Registration is also open at the “early bird” rate until 31 December 2023. … [Read more]
A large series of patients with VCP gene-related myopathy studied with muscle imaging
An international consortium of clinicians coordinated by the Barcelona Neuromuscular Center has published the results of the analysis of nuclear magnetic resonance (MRI) images acquired in patients with VCP myopathy, a myopathy characterized by inclusion bodies and, inconstantly, Paget’s disease and/or fronto-temporal dementia : the images of 80 patients from the large international cohort of … [Read more]
“Fête de la Science” from 9 to 16 October
This year’s “Fête de la Science” will take place from 9 to 16 October in France. Created in 1991, this event is organized every year by the French Ministry of Higher Education and Research, to enable everyone to discover the world of science, and meet the men and women who make today’s science possible. For … [Read more]
