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Spinal Muscular Atrophy News

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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Biogen & Ionis to collaborate again on treatments for SMA

Biogen and Ionis Pharmaceuticals have announced that they have entered into a new collaboration to identify new antisense oligonucleotide drugs for the treatment of SMA. These companies were responsible for the development and commercialisation of SPINRAZA (nusinersen), the first approved treatment for SMA. SPINRAZA is also an antisense oligonucleotide, which acts by increasing the amount…

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AVEXIS announced Phase 1 clinical trial of AVXS-101 for SMA Type 2

Avexis has been granted authorisation to start Phase 1 clinical trial of AVXS-101 for patients with SMA Type 2 by the U.S. Food and Drug Administration (FDA), based on review of data submitted. The company plans to initiate this trial immediately. The open-label, dose-comparison, multi-center Phase 1 trial named STRONG, is designed to evaluate the…

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New research into the role of the SMN protein

Researchers co-funded by The SMA Trust and Muscular Dystrophy UK have discovered a new role for the SMN protein, which is reduced in people with spinal muscular atrophy (SMA). These findings improve our understanding of SMA and will help to develop future treatments.     What were the researchers aiming to do? SMA is a…

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