News

Spinal Muscular Atrophy News

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…

READ ARTICLE

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…

READ ARTICLE

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…

READ ARTICLE

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…

READ ARTICLE

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…

READ ARTICLE

Cytokinetics recruits second cohort for Phase 2 trials

Cytokinetics announced this week that they have started recruitment for the second cohort of the Phase 2 clinical trial of CK-2127107 in patients with SMA in Canada and the USA. This clinical trial is designed to assess the effect of CK-2127107, a next-generation fast skeletal troponin activator (FSTA), on multiple measures of muscle function in…

READ ARTICLE

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…

READ ARTICLE