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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The National Institute for Health and Care Excellence’s (NICE) have published draft guidance ‒…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…
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We reported recently on a key step in the US Food and Drug Administration’s (FDA) pathway for…
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Pompe disease is a chronic, rare, genetic, progressive metabolic disease which results in severe…
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UPDATE: On 24 May 2023, Roche published an update in which they stated that the FDA's final…
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We reported last year that Fulcrum Therapeutics announced that it will be starting a phase 3…
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Read NICE's guidance here.
MDUK has been leading the fight for access to the treatment since 2016…
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We advise that you contact your clinical team if you have any concerns.
Update from NHS England…