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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On 30 September 2022, NICE published its draft guidance on Translarna (also known as ataluren).…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…
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Changing Places toilets are specifically designed and equipped for people with significant…
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We were joined by Neuromuscular Care Advisors Pam Appleton, from St Georges University Hospital,…
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Following the completion of the Phase 2 clinical trial, Fulcrum therapeutics recruited its first…
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Integrated Care Systems (ICSs) are now official statutory bodies, as of today, 1 July 2022. In…