Spinal Muscular Atrophy News

Improving energy production could protect motor neurons from SMA

SMA Trust-funded researchers have found that improving energy production in certain motor neurons with SMA could potentially protect them from damage. This could lead to the development of new treatments. What were the researchers aiming to find out? SMA is a condition affecting motor neurons (the nerves that connect the spinal cord to muscle), causing…


Biogen statement following disclosure of EU list price for nusinersen

On Thursday 1st June 2017, Biogen announced that the European Commission has granted marketing authorisation to nusinersen for the treatment of 5q Spinal Muscular Atrophy (SMA). The approval of nusinersen was based on positive results from multiple clinical studies in more than 260 patients treated with nusinersen, and has demonstrated breakthrough clinical value, making a…


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…


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…


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…


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…