These are exciting times, with fresh news about research developments appearing every week.
Find out about the latest research news below and also visit our Breaking News in Research page.
New stem cell therapy shows promise in Duchenne trial
Capricor Therapeutics has recently published the final results from its HOPE-Duchenne clinical trial. This study evaluated the safety and efficacy of a cell therapy called CAP-1002
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Highlights of the 2019 Neuromuscular Translational Research Conference
11/04/2019The 12th UK Neuromuscular Translational Research Conference was held in Newcastle on 4-5 April 2019.
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Results from gene therapy trial for limb girdle muscular dystrophy type 2D
02/04/2019One adult and six children with LGMD2D were treated with the gene therapy MYO-102. The treatment was safely delivered to their muscles and a new clinical trial is being planned.
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New study on the impact of steroids on bone health in Duchenne
26/03/2019A new study has found that boys with Duchenne muscular dystrophy on certain steroid regimes have a higher fracture risk than others.
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World-leading neurologist to join team at MDUK Oxford Neuromuscular Centre
25/03/2019Acclaimed clinical researcher, Professor Laurent Servais, has been appointed Professor of Paediatric Neuromuscular Diseases at the MDUK Oxford Neuromuscular Centre.
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Have your say on research into mitochondrial disease
21/03/2019The aim of the Mitochondrial Disease Priority Setting Partnership (PSP) is to find the most important unanswered questions about mitochondrial diseases.
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Duchenne study brings new understanding of genome editing
13/03/2019Researchers identify a way to improve the efficiency of genome editing technology and boost dystrophin production in a mouse model of Duchenne muscular dystrophy.
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CMT drug receives Orphan Drug designation
05/03/2019Acceleron announced today that the US FDA has granted Orphan Drug designation to their drug ACE-083 for the treatment of patients with Charcot-Marie-Tooth disease (CMT).
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British Science Week 2019
04/03/2019This Friday marks the start of British Science Week, a celebration of science, technology, engineering and maths.
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Positive results from LGMD gene therapy trial
28/02/2019Sarepta Therapeutics has announced preliminary results from the MYO-101 trial.
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Repurposed drug could be beneficial for OPMD
26/02/2019A new study has found that a drug used to treat high blood pressure could be beneficial for the treatment of oculopharyngeal muscular dystrophy (OPMD).