News

The purpose of SMA Europe is to provide a framework to stimulate collaboration and accelerate translational research pathways in SMA and promote patient care.

  1. Roche has provided an update on Olesoxime and RG7916

    Roche has provided an update on the development of neuroprotective agent Olesoxime and small molecule RG7916, which acts on the SMN2 gene. This updates includes information on which trials are currently recruiting, what the eligibility criteria are and how to participate in a trial as well as information on trial design. You can also find […]

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  2. 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 […]

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  3. 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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  4. 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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