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The news follows on from Italfarmaco Group announcing results from the EPIDYS clinical trial…
Blog
Duchenne muscular dystrophy (DMD) is a condition caused by genetic changes in the dystrophin gene…
Blog
A career in science and muscular dystrophy
I’ve always had a brain for science as opposed to…
News
We are supporting this process as one of two ‘Patient Experts’, and our Director of Care, Campaigns…
News
In September 2023 we shared news that the European Medicine Agency’s (EMA) human medicines…
News
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…
Blog
Understanding self-management support programmes
Self-management support (SMS) is a care programme…
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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In September we shared the news that NICE had published draft guidance on the use of the treatment…