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 CMT app launched
Pharnext is carrying out an observational study for people with Charcot-Marie Tooth disease using a new digital app called CMT&Me.
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FDA fast tracks SMA gene therapy
04/12/2018Novartis has announced that the US Food and Drug Administration (FDA) has accepted its marketing application for AVXS-101 for the treatment of SMA type 1.
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New FSHD mouse model treated with gene therapy
26/11/2018A recent study has demonstrated the value of a new mouse model of facioscapulohumeral muscular dystrophy (FSHD).
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Mitochondrial myopathy trial recruiting in the UK
15/11/2018Stealth BioTherapeutics has initiated a phase 3 trial to test elamipretide in individuals with primary mitochondrial myopathy.
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Care recommendations for myotonic dystrophy type 1 published
07/11/2018An international team of experts have published consensus-based care recommendations to help clinicians deliver high standards of care to adults with myotonic dystrophy 1.
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US charity receives funding for GNE myopathy gene therapy
25/10/2018The US based charity, Neuromuscular Disease Foundation, has been awarded a challenge grant to support gene therapy research for individuals with GNE myopathy.
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SMA gene therapy seeks marketing approval
19/10/2018AveXis has announced it is seeking marketing approval for its gene therapy, AVXS-101, in Europe, United States and Japan for infants with SMA type 1.
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Update on UNITE-DMD gene therapy trial
18/10/2018Work has begun in Professor George Dickson’s lab to refine the gene therapy product.
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CMT gene therapy trial to start in 2019
15/10/2018Sarepta Therapeutics has announced that it will be working with Dr Zarife Sahenk from Nationwide Children’s Hospital in the United States to develop a gene therapy for CMT.
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PTC announces long-term data on Duchenne drug
08/10/2018PTC Therapeutics recently presented positive preliminary data on the long-term clinical benefit of Translarna at the World Muscle Society conference.
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Update from X-MTM gene therapy trial
05/10/2018Today at the World Muscle Society conference, Audentes Therapeutics announced new data from its ASPIRO study testing a gene therapy for X-linked myotubular myopathy.