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What is spinal muscular atrophy?
Spinal muscular atrophy (SMA) is a rare muscle-wasting condition…
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1,147 people living with FSHD took part in the Europewide study, led by FSHD Europe, to identify…
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Back again, I thought with a smile on my face as the train pulled into the Liverpool Lime Street…
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Muscles contract so that we can move our bodies, breathe, swallow etc. Several muscle conditions…
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Read NICE's guidance here.
MDUK has been leading the fight for access to the treatment since 2016…
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We advise that you contact your clinical team if you have any concerns.
Update from NHS England…
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A better understanding the root cause behind this condition will in time lead to more targeted and…
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Duchenne muscular dystrophy (DMD) is caused by genetic changes that prevent the body from producing…
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The Phase 3 SAPPHIRE study aims to assess the safety and effectiveness of apitegromab used in…
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Facioscapulohumeral muscular dystrophy (FSHD) is the third most common inherited muscular dystrophy…