Breaking: Update on Translarna NHS treatment for Duchenne muscular dystrophy
The 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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Wonder Girl Carmela to star in superhero children’s book
05/05/2021‘Wonder Girl Carmela and Tinker the Stinker’ centres on the exciting adventures of Carmela Chillery-Watson and her therapy dog Tinker, and was written by her mum Lucy.
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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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More people eligible for access to Spinraza following NICE review of MAA data
04/05/2021More people eligible for access to Spinraza following NICE MAA review.
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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)
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Manifesto for Muscles in Scotland and Wales
21/04/2021Ahead of the upcoming elections for the Scottish Parliament and Welsh Parliament on Thursday 6 May, we have put together a Manifesto for Muscles for candidates to support.
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Breaking: SMC accepts Translarna for Duchenne muscular dystrophy treatment
12/04/2021A drug that can treat an underlying genetic cause of a muscle-wasting condition has been accepted for use on NHSScotland, it was announced today.