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Spinal Muscular Atrophy News

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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Infants, teens and young adults treated with Spinraza show improved motor function

New Data presented at the Muscular Dystrophy Association’s clinical conference show benefit in motor function for infants, teens and young adults treated with Spinraza™ (nusinersen). The NURTURE study results reported that all study participants (25 infants) were alive and experienced improved motor function at the time of reporting, compared to a decline seen in SMA…

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Biogen releases statement on CHERISH data

Following a request from SMA Europe, Biogen sent a community update on the end of study results from CHERISH, the Phase 3 trial evaluating nusinersen for the treatment of individuals with later-onset SMA, which have now been published in the New England Journal of Medicine (NEJM). The full manuscript entitled, “Nusinersen Versus Sham Control in…

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SMA Europe’s International Scientific Congress on Spinal Muscular Atrophy

SMA Europe held its first International Scientific Congress on spinal muscular atrophy in Kraków, Poland between January 25th and January 27th, 2018.   Dr. Ewout Groen, our research communications adviser, reports on this exciting event. A record number of 463 delegates attended a packed programme that included 35 oral presentations across 7 sessions, 75 poster…

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New trials planned for gene therapy product AVXS-101

AveXis announced its plans to expand the clinical development program for the company’s gene therapy candidate, AVXS-101, for the treatment of SMA. In addition to the ongoing pivotal trial in SMA Type 1 (STR1VE) and the ongoing Phase 1 trial in SMA Type 2 (STRONG), the company plans to initiate three studies to further evaluate…

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Managed Access Agreement for Spinraza – Biogen Update

A Managed Access Agreement (MAA) for Spinraza has been confirmed as currently in discussion by the manufacturer, Biogen. The MAA for Spinraza is being explored by Biogen, NHS England and NICE (National Institute for Health and Care Excellence), whilst the longer term funding of the treatment is considered under the Single Technology Appraisal (STA) process, by…

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NICE Spinraza appraisal – help us work towards quicker access

NICE, The National Institute for Health and Care Excellence has confirmed it has started the appraisal of Spinraza following a frustrating delay, which has been partially down to uncertainty about how the treatment will be reviewed for funding by the NHS. The treatment will now be being assessed for all types of SMA, with Spinraza…

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