Latest News: Research
15 November 2017 / Posted in: Research
The 22nd International Annual Congress of the World Muscle Society (WMS) was held October 3rd-7th 2017 in Saint Malo, France. While SMA Support UK did not attend, Biogen have uploaded three posters to their repository that were presented at the meeting.
Clinicians and Charities Make Further Appeal to NHS England to Widen Scope of SMA Type 1 Nusinersen EAP
30 October 2017 / Posted in: Research
SMA Support UK has backed the call led by Professor Francesco Muntoni not to limit the treatment only to infants with two SMN2 copy numbers.
26 October 2017 / Posted in: Research
The All Party Parliamentary Group (APPG) on Rare, Genetic and Undiagnosed Conditions has asked Genetic Alliance UK to lead work to propose a solution.
18 October 2017 / Posted in: Research
We share the frustrations of the SMA Community that there has still been no announcement from NICE about whether it will assess the treatment for continued and wider provision of nusinersen by the NHS. Here is an update of what SMA Support UK is doing.
12 October 2017 / Posted in: Research
The Food and Drug Administration (FDA) has granted permission to AveXis to begin their planned US pivotal trial of their gene therapy drug for SMA, AVXS-101. Avexis say that they are to immediately begin the trial.
11 October 2017 / Posted in: Research
Muscular Dystrophy UK are campaigning for faster access to specialist treatments, including Spinraza for SMA. You can find out more and support the 'FastTrack' Campaign here.
29 September 2017 / Posted in: Research
The request to include those with more than two SMN2 copies and over 7 months of age in the SMA Type 1 nusinersen EAP is not yet successful. We are still waiting to hear from NICE about next steps.
27 September 2017 / Posted in: Research
Novartis has released the following community update for the LMI070 (branaplam) clinical trial.
19 September 2017 / Posted in: Research
Roche have provided an update on clinical trials of their SMN2 splice-modifying drug RG7916
30 August 2017 / Posted in: Research
You can now read the full workshop report written by the clinical researchers and patient representatives from Europe and the US who met last year.