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Read the latest news, browse other people's stories and find blogs about experiences you relate to. You can also read our publications like Target MD, and find our most recent press releases.

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Research lab image of equipment
Research reveals new cause of muscle weakening in spinal muscular atrophy
What is spinal muscular atrophy? Spinal muscular atrophy (SMA) is a rare muscle-wasting condition…
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Sheila Hawkins
To coincide with Rare Disease Day FSHD Patients across Europe say what they want from clinical trials
1,147 people living with FSHD took part in the Europewide study, led by FSHD Europe, to identify…
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Researcher working within a research lab
“If you are constantly trying to cure sneezing, which is a symptom of a cold, then you won’t get very far” - my visit to Liverpool Universities
Back again, I thought with a smile on my face as the train pulled into the Liverpool Lime Street…
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Research image of a scientist in orange gloves working with a beaker
Researchers find potential key to treatment for myopathies in the pocket of an old muscle protein
Muscles contract so that we can move our bodies, breathe, swallow etc. Several muscle conditions…
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Breaking News
NICE publishes final guidance recommending access to Duchenne muscular dystrophy treatment Translarna
Read NICE's guidance here. MDUK has been leading the fight for access to the treatment since 2016…
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Breaking News
Temporary change to eligibility for SMA treatment Zolgensma® in England
We advise that you contact your clinical team if you have any concerns. Update from NHS England…
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Image of equipment in a research lab
New research sheds light on the cause of myotonic dystrophy type 1
A better understanding the root cause behind this condition will in time lead to more targeted and…
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Close up image of a researcher's hand, adding a liquid into a tube
New research may help predict the severity and progression of Duchenne muscular dystrophy
Duchenne muscular dystrophy (DMD) is caused by genetic changes that prevent the body from producing…
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News
Half and half image, on the right is a headshot of Dr Kate Adcock on an orange background. On the left, is white text on an orange background saying '"It's encouraging to see this trial combining two SMA treatments, which could prove beneficial for people living with the condition." Dr Kate Adcock, Director of Research, Muscular Dystrophy UK'
First UK clinical trial site for new combined SMA treatment
The Phase 3 SAPPHIRE study aims to assess the safety and effectiveness of apitegromab used in…
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An image of a female researcher, sitting in a lab, looking through a microscope. The image is taken from an angle, facing the right, front side of the researcher.
Targeting the cell’s energy system as a possible treatment for FSHD
Facioscapulohumeral muscular dystrophy (FSHD) is the third most common inherited muscular dystrophy…
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