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Spinal Muscular Atrophy News

Biogen to close Spinraza EAP from November

Following the announcement that Spinraza/nusinersen will not be funded on the NHS, Biogen the manufacturer announced late yesterday that they will be closing the Expanded Access Programme (EAP) as of 1st November. The EAP is a temporary compassionate use arrangement with the NHS, which has enabled access to Spinraza by 80 Type 1 babies in…

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Biogen Response to NICE Spinraza Announcement

Biogen have issued a press release following yesterdays disappointing announcement by NICE (National Institute for Health and Care Excellence), recommending that Spinraza/nusinersen not be funded, on the NHS. Their response acknowledges the difficulties for rare disease treatment approval, within the current assessment criteria used by NICE and demonstrates a commitment to working with both NICE…

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Spinraza not to be funded – NICE Announce

Spinraza (Nusinersen) will not be recommended for funding by the NHS, is the devastating decision announced today by NICE (National Institute for Health and Care Excellence). One glimmer of hope, however, is that it does leave the door open for a Managed Access Agreement. NICE’s consultation paper was released this morning with the devastating news…

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We are merging with SMA Support UK

Thank you to all of you who took the time to respond to our merger consultation survey – 76% of you considered the proposal to join together as ‘excellent’ and 22.5% of you thought it was ‘good’. All feedback received has been considered by both Boards and will help to inform the next steps, but…

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Biogen adds potential side-effect to Spinraza label

The European Spinraza label (packaging and product information leaflet) will soon include hydrocephalus as a potential side-effect. This is a precautionary measure and, as yet, there is no evidence that hydrocephalus has been caused by nusinersen. No cases of hydrocephalus were observed in clinical trials. Biogen has provided the following community update Download Biogen label…

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Biogen update on European Spinraza access

Whilst the process for determining whether Spinraza will be funded in England continues, Biogen, the manufacturer, issued a community announcement with updates as to availability across Europe, which can be read here. .

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SMA Trust & SMA Support UK Proposed Merger

The SMA Trust and SMA Support UK have announced an intention to merge, requesting thoughts and feedback from our friends, supporters and the SMA community. Having worked together increasingly closely over the past few years, we feel we can achieve more for the SMA community and faster, by coming together formally. You can read the full…

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Roche Sunfish Trial to exclude UK

Roche update: Sunfish trial not going ahead in the UK We have received the following update from Roche about the SUNFISH study, investigating RG7916 in people with SMA Type 2 and 3. “In the last few weeks we have seen a rapid increase of recruitment into the SUNFISH study. All available screening slots in SUNFISH…

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New Scottish Pathway for Access to Rare Disease Medicines

Scottish Health Secretary outlines new pathway for medicines for rare conditions. Patients with very rare diseases could get faster access to new treatments following a revised decision making process. The Scottish Government is introducing a new definition of ‘ultra-orphan medicines’ that can treat very rare conditions affecting fewer than 1 in 50,000 people – around…

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