Duchenne muscular dystrophy
RSS feedSustainable cardioprotection with utrophin in DMD
Scientists have evaluated a gene therapy based on micro-utrophin, a protein similar to dystrophin, which is deficient in Duchenne muscular dystrophy (DMD). This therapy was administered to mice models of the disease, exposed to pharmacological and exercise-related stress. The therapy provided protection against cardiac damage, as evidenced in particular by a decrease in troponin levels. … [Read more]
Unfavorable opinion on marketing authorisation and suspension of clinical trials in Europe for Elevidys in DMD
Over the summer, the European Medicines Agency (EMA) issued an unfavourable opinion on the conditional marketing authorisation of Elevidys, a microdystrophin gene therapy for ambulatory patients aged 3 to 7 years with Duchenne muscular dystrophy (DMD). This opinion, which must be ratified by the European Commission, is based on insufficient data on the treatment’s efficacy … [Read more]
Review of current and emerging therapeutic strategies in DMD
Duchenne muscular dystrophy (DMD) is a serious, progressive genetic disorder. It is caused by mutations in the DMD gene that result in the absence of dystrophin, an essential structural protein of the sarcolemma. This weakens the sarcolemmal membrane and makes it highly sensitive to mechanical stress. The heart muscle suffers degenerative alterations similar to those … [Read more]
Advances in Duchenne muscular dystrophy and Becker muscular dystrophy – 2025
This document presents a selection of Duchenne muscular dystrophy and Becker muscular dystrophy research news stories from the past year (ongoing observational studies and clinical trials, scientific and medical publications, etc.). Access the document Advances in Duchenne muscular dystrophy and Becker muscular dystrophy – 2025
UK recommendations to improve orthopedic care in DMD
A group of British experts has formulated recommendations, approved by the British Society for Children’s Orthopaedic Surgery, aimed at harmonizing and improving orthopedic care for children and young adults with Duchenne muscular dystrophy (DMD). Non-urgent care should be provided in specialized centers, but fractures can be treated by local trauma units if sufficient medical and … [Read more]
Research into biomarkers to differentiate Becker and Duchenne muscular dystrophies
Swedish and Dutch researchers used mass spectrometry to try to identify proteomic profiles that would distinguish between the two most common types of dystrophinopathy, namely Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD): 34 patients with BMD and 19 with DMD were included in the study, which lasted three years and consisted of collecting … [Read more]
A cardiac micro-pump useful in cases of decompensated cardiomyopathy in DMD
American researchers report the successful implantation of a temporary Impella 5.5 endocavitary micro–pump developedby Abiomed: the patient was a 14–year–old boy with Duchenne muscular dystrophy, he had been transferred for treatment of cardiogenic shock in the context of decompensation of his cardiomyopathy. the implantable device was inserted through the right axillary artery into the left … [Read more]
Gene therapy and DMD: a possible link with cardiac inflammation?
Following the serious side effects observed during trials of micro–dystrophin gene therapy using an AAV viral vector in Duchenne muscular dystrophy (DMD), researchers at the Institute of Myology undertook to investigate the mechanismsinvolved in greater depth: a transgenic mouse with a double knockout for dystrophin and utrophin was used as an experimental model, treated with … [Read more]
DMD: Canakinumab reduces certain blood markers but not IL1b
Canakinumab (Ilaris®) is a monoclonal antibody that neutralises interleukin 1 beta (IL1β), an inflammatory marker that is highly expressed in Duchenne muscular dystrophy. It is an immunosuppressant that is already on the market. A US phase I/II trial evaluated the efficacy of 2 mg/kg of canakinumab in three boys with DMD (two aged 4 and … [Read more]
DMD: launch of the pivotal phase of Généthon’s gene therapy trial
The results of the gene therapy trial (GNT0004) conducted by Généthon for Duchenne muscular dystrophy were presented on 17 May at the 2025 conference of the American Society of Gene & Cell Therapy (ASGCT). They demonstrate stabilisation of motor function and a significant and sustained reduction in CPK levels in patients treated at the effective … [Read more]
