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Spinal Muscular Atrophy News

nusinersen (Spinraza) – a milestone but not the end game

STATEMENT FROM THE SMA TRUST   The news from the US of the FDA’s approval of Spinraza, the first treatment for SMA, has changed the SMA landscape for ever.  Accelerated approval is also being sought from The European Medicines Agency (EMA) for a European licence and then it will be up to individual countries to appraise…

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New phase 3 ENDEAR data presented by Biogen

New phase 3 ENDEAR data presented by Biogen at the British Paediatric Neurology Association (BPNA) annual conference in Cambridge last week, show that SPINRAZA™ (nusinersen) significantly reduces the risk of death or permanent ventilation in SPINRAZA-treated infants with SMA compared to untreated infants. In August 2016, Biogen reported that ENDEAR met its pre-specified primary endpoint at…

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NICE Scoping Workshop – Joint charities update

Joint charities update following the NICE Scoping workshop to look at the draft remit and scope for the proposed Technology Appraisal, nusinersen for treating spinal muscular atrophy. Representatives from MDUK, The SMA Trust, SMA Support UK and Genetic Alliance attended a meeting with the National Institute of Health and Care Excellence (NICE) on 13th January,…

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The SMA Trust, MD UK & SMA Support UK write to the EMA

The SMA Trust, in partnership with Muscular Dystrophy UK and SMA Support UK have written to the EMA requesting assistance in ensuring nusinersen (Spinraza) is made available at the earliest possible time for the treatment of spinal muscular atrophy in patients in Europe. We have written to ask what steps the EMA can take to…

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Spinraza (Nusinersen) approved by FDA in the US

Joint statement from The SMA Trust, Muscular Dystrophy UK and SMA Support UK: Just before Christmas, the United States Food and Drug Administration (FDA) announced that it has approved Spinraza as a treatment for SMA. Spinraza is the brand name for the drug, nusinersen. This approval is for a broad licence, meaning that Spinraza can…

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SMA Trust Spinraza update

We’re delighted with the news of the FDA’s approval of Spinraza in the US (as we reported just before Christmas) and, like everyone else, very much hope for EMA approval in a few month’s time, followed by NICE and other bodies involved in approving new drugs in the UK . We’ve been working closely with…

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Roche provides community update on RG7916 for SMA

Roche provides a community update on the investigational product RG7916, which they are developing together with PTC Therapeutics and the SMA Foundation in the US. This compound is an orally-administered SMN2 splicing modifier, for which there are 3 studies: Firefish, Sunfish and Jewelfish. All include different populations. They are pleased to report that Sunfish (Type…

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Spinraza (Nusinersen) approved by the FDA

Yesterday, the FDA announced that it has approved Spinraza (Nusinersen) to treat spinal muscular atrophy, making it the first-ever FDA-approved therapy for SMA, a product which addresses the underlying genetic cause of the condition by making the back-up gene, SMN2, produce more functional SMN protein. This milestone has been achieved as a result of the…

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Update on Nusinersen Expanded Access Programme in the UK

Update on progress towards an Expanded Access Programme of the ENDEAR trial of nusinersen which treats eligible infants with Spinal Muscular Atrophy Type 1   Joint statement from The SMA Trust, SMA Support UK and Muscular Dystrophy UK.  Families may recall reading the last update in September 2016 which was released by the Principal Investigators from…

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The Lancet publishes Nusinersen (Spinraza) Phase 2 Trial Results

Results from a phase 2 clinical trial of the Ionis Pharmaceuticals gene therapy drug, nusinersen (now marketed as Spinraza), have been published in the renowned medical journal The Lancet. Initiated in 2013, this trial was an open-label study designed to assess the safety, tolerability, pharmacokinetics, and efficacy of repeated spinal cord injections of nusinersen, an…

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