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NICE likely to recommend mexiletine for treating the symptoms of myotonia in adults with non-…
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We were joined by Prof Mary Reilly (Professor of clinical Neurology at UCL) who spoke about the…
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To mark Becker awareness week, we were joined in our BMD seminar by researcher Federica Montanaro,…
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There are currently no approved drugs for Charcot-Marie-Tooth disease (CMT), but clinical trials…
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Orphan drug status for treatment of myotonic dystrophy type 1
With no cures for myotonic…
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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…