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We are supporting this process as one of two ‘Patient Experts’, and our Director of Care, Campaigns…
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In September 2023 we shared news that the European Medicine Agency’s (EMA) human medicines…
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In January 2019, we partnered with the University of Oxford to form the MDUK Oxford Neuromuscular…
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Limb girdle muscular dystrophy 2E/R4, also known as beta sarcoglyanopathy, is a progressive genetic…
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XLMTM and liver disease
Over the past few years, it’s become clear that some people with XLMTM can…
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In September we shared the news that NICE had published draft guidance on the use of the treatment…
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UPDATE (3 November 2023):
A webinar regarding the EMBARK study results for families took place…
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Calling all researchers – we’re pleased to announce the 2024 Grant Round for research proposals…
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The National Institute for Health and Care Excellence’s (NICE) have published draft guidance ‒…
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Our new research grants cover a variety of different muscle-wasting conditions, such as ADSSL1…