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Approval of gene therapy SRP-9001 in the USA ‒ a significant milestone in the treatment of Duchenne muscular dystrophy
We reported recently on a key step in the US Food and Drug Administration’s (FDA) pathway for…
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Breaking News
NICE recommend new treatment option for late-onset Pompe disease
Pompe disease is a chronic, rare, genetic, progressive metabolic disease which results in severe…
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News-Update
US FDA advisory committee votes on Sarepta SRP-9001 gene therapy to treat Duchenne muscular dystrophy ahead of a FDA’s final decision
UPDATE: On 24 May 2023, Roche published an update in which they stated that the FDA's final…
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News-Update
UK sites are now recruiting people with FSHD to take part in a clinical trial
We reported last year that Fulcrum Therapeutics announced that it will be starting a phase 3…
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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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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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Research strategy blog image
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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Research strategy blog image
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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