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On Monday 23 January, we let you know that NHS England had made a decision that made it impossible…
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On 24 January 2023, after this article was originally published, we were informed by NHS England…
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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…
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In the Chancellor’s Autumn Statement on 17 November 2022, we were pleased to hear commitments to…
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The Phase 3 SAPPHIRE study aims to assess the safety and effectiveness of apitegromab used in…
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On 30 September 2022, NICE published its draft guidance on Translarna (also known as ataluren).…
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As part of Spinal Muscular Atrophy Awareness Month, we split up our past SMA Muscles Matter videos…
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Within Avalglucosidase alfa’s approval process, MDUK took part in a NICE Scoping Workshop in…
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We’re delighted to announce that a new treatment for Pompe disease has been recommended for use by…