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.
Announcing the establishment of the FSHD European Trial Network
This spring, FSHD Europe and FSHD researchers from across Europe, including the UK, organised two workshops to help establish a European FSHD Trial Network.
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NICE recommends Zolgensma for use in England
04/06/2021NICE recommends Zolgensma for use in England
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NICE to consult on decision not to recommend SMA treatment Risdiplam on NHS in England
02/06/2021We're disappointed to say that, following its appraisal, NICE is not recommending the SMA treatment Risdiplam for use on the NHS.
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SMA Type 1 gene therapy administered for first time in England on the NHS
01/06/2021We’re delighted to hear the news today that the first baby in England has been treated with Zolgensma, a potentially life-changing gene therapy for SMA Type 1.
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Breaking: Update on Translarna NHS treatment for Duchenne muscular dystrophy
13/05/2021The MAA has been extended until January 2023.
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First UK participant enrolled into Pfizer’s Duchenne muscular dystrophy gene therapy trial.
12/05/2021The first UK participant has been enrolled in Pfizer's gene therapy trial for DMD.
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Novartis announces plans for new Zolgensma gene therapy trial for SMA
06/05/2021Last month the pharmaceutical company Novartis announced plans to start a new international clinical trial of the spinal muscular atrophy (SMA) gene therapy Zolgensma.
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Sarepta shares update from DMD exon 51-skipping clinical trial MOMENTUM
06/05/2021Sarepta Therapeutics has released an update from its MOMENTUM study investigating SRP-5051 for the treatment of boys with Duchenne muscular dystrophy
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International gene therapy trial starts in France
05/05/2021We’re delighted to tell you that the first boy with Duchenne muscular dystrophy has been dosed as part of a clinical trial to study a gene therapy for the condition.
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Improving the delivery of potential treatments for muscle-wasting conditions
23/04/2021Read more about research we fund to develop and improve the delivery of potential therapies for muscle-wasting conditions.
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Results from Orphanzyme inclusion body myositis (IBM) clinical trial
23/04/2021Orphanzyme announced negative results from its Phase 2/3 clinical trial testing arimoclomol for the treatment of inclusion body myositis (IBM)