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Spinal Muscular Atrophy News

Ride Scorpion 2017

On 6th May, through the windy yet dry weather, keen and novice cyclers took on the third year of our Ride Scorpion event. It was a lovely day and a great atmosphere. Everybody finished and had a great time enjoying some of the best views that the Cotswolds can offer, followed by food and entertainment…

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NHS England meeting on Spinraza – Update

A meeting has recently taken place where SMA Support UK and Muscular Dystrophy UK, representing SMA charities, met with the Clinical Director of Specialised Commissioning at NHS England, James Palmer, for urgent talks on the Expanded Access Programme for Spinraza to treat infants with SMA Type 1. They were also joined by representatives from the…

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Spinraza improves motor function in children with later-onset SMA

Biogen presented data from the end of the Phase 3 study on Spinraza™ (CHERISH) at a plenary session of the American Academy of Neurology (AAN) annual meeting in Boston on 24th April 2017. This data demonstrated a highly statistically significant and clinically meaningful improvement in motor function in children with later-onset (most likely to develop Type 2…

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SMA Europe Annual Meetings 2017

SMA Europe’s annual meetings this year were hosted by the Dutch neuromuscular disease association, VSN (Vereniging Spierziekten Nederland) in Utrecht between 6th & 8th April 2017. The programme included: joint and individual meetings with the main pharmaceutical companies involved in the development of therapies for SMA; the review of 21 SMA research grant applications by…

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The SMA Trust team is on the move!

Dear Supporter, The team has moved move! Our new contact details are: 3 Wychwood Court Cotswold Business Village Moreton in Marsh GL56 0JQ Tel: 01608 801020. Our old number (01789 801155) has been diverted indefinitely. Please note that we are still experiencing disruptions to both our IT and phone systems. We apologise for any inconvenience this may…

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Biogen EAP Update – 13 April 2017

Dear members of the SMA community, In response to your request for information, we would like to provide you with an update on Biogen’s global Expanded Access Program (EAP). Based on the high unmet need in SMA, and to bridge the gap between the closure of the shamcontrolled phase 3 ENDEAR study and regulatory approval…

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AveXis Phase 1 Trial of AVXS-101 for SMA and plans for Europe

AveXis reported topline results from the Phase 1 trial of its gene therapy product AVXS-101 for SMA Type 1. Find out how this product works here. “The completion of our Phase 1 clinical study of AVXS-101, the first ever gene therapy studied for the treatment of SMA Type 1, is an exciting and eagerly awaited…

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