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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…
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The Phase 3 SAPPHIRE study aims to assess the safety and effectiveness of apitegromab used in…
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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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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…