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Within Avalglucosidase alfa’s approval process, MDUK took part in a NICE Scoping Workshop in…
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We’re delighted to announce that a new treatment for Pompe disease has been recommended for use by…
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We were joined by Neuromuscular Care Advisors Pam Appleton, from St Georges University Hospital,…
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Following the completion of the Phase 2 clinical trial, Fulcrum therapeutics recruited its first…
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What is Duchenne muscular dystrophy? Duchenne muscular dystrophy is a serious genetic condition…
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We are delighted to announce that Muscular Dystrophy UK (MDUK) is partnering with the charity…
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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…