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Spinal Muscular Atrophy News

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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NICE Committee Meets Re: Spinraza Access

The SMA Trust attended the NICE (National Institute of Health and Care Excellence) Committee meeting relating to Spinraza access in England, on Wednesday 27th June, in Manchester. The Committee considered clinical and economic evidence, supporting the funding of the treatment, as well as listening to powerful accounts from patient representatives. Thanks and well done to…

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Roche ends Olesoxime development

News that Roche has made the difficult decision to end the development of neuro-protective compound Olesoxime, was announced on 30th May. The OLEOS study, evaluating the safety and effectiveness of Olesoxime, was started in 2016. Since then, ongoing discussions with Health Authorities (FDA and EMA) and SMA experts were held and the company has continued…

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Biogen response to letter re: Scottish Spinraza Access

Following, the SMA partner organisations collective letter to Biogen management to request a review of pricing and resubmission to The Scottish Medicines Consortium, in order to enable further progress and access to Spinraza for SMA Types 2 and 3, this response was received. .

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Scottish Spinraza Access – letter to Biogen

Following, The Scottish Medicines Consortium Committee announcement of their decision to accept Spinraza for restricted use in NHS Scotland in infantile onset Type I SMA only, ourselves and our SMA partner organisations have collectively written to Biogen’s management to request a review of pricing and resubmission in order to enable further progress and access for…

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Scottish Access to Spinraza – announcement

Today, The Scottish Medicines Consortium Committee announced their decision to accept Spinraza for restricted use in NHS Scotland in infantile onset Type I SMA.  Whilst we recognise this is a positive step forward and a lifeline for these children and families, it is hugely disappointing that children, young people and adults with Type 2 and…

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Scottish Access to Spinraza – PACE Meeting

SMC PACE Meeting – powerful patient testimonies convey the urgent need for Spinraza in Scotland On 13th March we attended a special PACE (Patient and Clinician Engagement) meeting at the Scottish Medicines Consortium in Glasgow. This meeting formed part of their appraisal process for Spinraza and is an additional stage that can be used to gain…

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