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We were joined by Occupational Therapists Colin Jones and Bea Moon, parent Gerry McMenemy and…
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We were joined in our SMA seminar by Consultant Neurologist Dr Channa Hewamadduma, NICE…
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Tens of thousands more people in the UK than previously thought are living with rare, muscle-…
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Data from a study of godolirsen has demonstrated the long-term safety of the treatment and showed…
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We were joined by Dr Scott Davidson & Sr Alison Clarke from Queen Elizabeth University Hospital…
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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…