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.
Highlights from international myotonic dystrophy conference
Our Research Communications Manager, Dr Jenny Sharpe, recently attended the twelfth meeting of the International Myotonic Dystrophy Consortium (IDMC) in Gothenburg, Sweden.
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Highlights from the 2019 FSH Society International Research Congress
15/07/2019The FSH Society International Research Congress was held in France, on 19-20 June 2019
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GNE myopathy registry goes live
03/07/2019The registry is once again live. If you haven’t yet signed up to it, you can do so now, and if you have signed up for it previously, you can go in and update your records.
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Blocking myostatin shows benefits in a mouse model of OPMD
10/06/2019Positive results found from blocking myostatin in a mouse model of OPMD. This demonstrates that treatments not targeting the genetic cause could still be beneficial.
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SMA gene therapy receives approval from US regulator
29/05/2019The US FDA has granted approval to Zolgensma, the first gene therapy for SMA. Although the approval process in Europe is separate to the US, it’s a major step forward.
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Santhera announces collaboration to develop CMD gene therapy
21/05/2019Santhera Pharmaceuticals announced that it has started a collaboration with the University of Basel to develop a gene therapy for LAMA2-deficient congenital muscular dystrophy.
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New MDUK-funded study investigating genetic tools to correct Collagen VI mutation
09/05/2019MDUK-funded researchers have corrected a common mutation causing Ullrich congenital muscular dystrophy in the laboratory.
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Sarepta acquires rights to sixth LGMD gene therapy
08/05/2019Sarepta Therapeutics announced today that it has signed an agreement with Nationwide Children’s Hospital (Ohio, USA) to develop a gene therapy for LGMD type 2A.
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Care recommendations for congenital myotonic dystrophy and myotonic dystrophy type 2 published
03/05/2019The first set of care recommendations for congenital and childhood-onset myotonic dystrophy type 1, and myotonic dystrophy type 2 have been published.
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Clinical trial for centronuclear myopathy given the green light in the UK
30/04/2019The drug company Dynacure has announced today that UK regulators have approved its UNITE-CNM phase I/II clinical trial.
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New activity study open to recruitment
30/04/2019To participate in the new study, you’ll simply need to live your life as usual and complete some questionnaires as well as wear an activity monitor for certain periods.