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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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Image: Image of genes Text: MDUK Muscles Matter 2022 Spinal muscular atrophy package
Spinal Muscular Atrophy (SMA) MDUK Muscles Matter - Package
As part of Spinal Muscular Atrophy Awareness Month, we split up our past SMA Muscles Matter videos…
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10 treatments either available or being appraised in major milestone for the community
Within Avalglucosidase alfa’s approval process, MDUK took part in a NICE Scoping Workshop in…
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New treatment for Pompe Disease recommended by NICE
We’re delighted to announce that a new treatment for Pompe disease has been recommended for use by…
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MDUK Muscles Matter 2022 - Friday 15 July round-up
We were joined by Neuromuscular Care Advisors Pam Appleton, from St Georges University Hospital,…
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Breaking News
First patient enrolled in phase 3 Losmapimod clinical trial for FSHD
Following the completion of the Phase 2 clinical trial, Fulcrum therapeutics recruited its first…
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Cell therapy as a possible approach to Duchenne muscular dystrophy treatment
What is Duchenne muscular dystrophy? Duchenne muscular dystrophy is a serious genetic condition…
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Neuromuscular Translational Research Conference 2022
MDUK Chair, Prof Mike Hanna and MDUK’s Director of Research, Dr Kate Adcock spoke at the event.…
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MDUK News Update
New £1m grant partnership with LifeArc into CMD research
We are delighted to announce that Muscular Dystrophy UK (MDUK) is partnering with the charity…
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MDUK Muscles Matter 2022 - Monday 21 March round-up
We were joined by Occupational Therapists Colin Jones and Bea Moon, parent Gerry McMenemy and…
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