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SMA UK, MDUK and TreatSMA have today written to the Scottish Medicines Consortium (SMC) to raise…
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This study is an extension of a phase 2 clinical trial in which 16 boys with Duchenne muscular…
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The results of the trial showed that people taking the drug have a slowing of disease progression…
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Risdiplam is an oral drug that increases SMN protein levels by targeting the SMN2 gene manufactured…
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Muscular Dystrophy UK is funding this research, looking into how dystrophin works in the heart and…
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Muscular Dystrophy UK welcomes this exciting news which was revealed earlier this week.
Detailed…
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Pfizer’s ongoing clinical trial is testing the efficacy and safety of the potential gene therapy…
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Muscular Dystrophy UK is very disappointed to learn that Mexiletine might not be recommended by…
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The Highly Specialised Service for Rare Mitochondrial Disorders in London has created a database to…