Latest News: Research
15 December 2017 / Posted in: Research
The Food and Drug Administration (FDA) has granted permission to AveXis to begin a second Phase 1 trial of their gene therapy drug, AVXS-101, with SMA Type 2 patients in the United States.
06 December 2017 / Posted in: Research
Catalyst Pharmaceuticals have announced that they are to investigate the potential of a drug called Firdapse as a treatment for people with SMA Type 3.
30 November 2017 / Posted in: Research
We have now received a further update and clarification that the Scottish Medicines Consortium will be assessing nusinersen for "pre-symptomatic infantile-onset and later-onset 5q SMA, but not for before birth or Adult Onset SMA".
22 November 2017 / Posted in: Research
Genetic Alliance voices the Rare Disease Community's concerns about the proposed changes to the NICE Technology Appraisal Programme.
17 November 2017 / Posted in: Research
Biogen’s SMA Community Update to SMA Europe covers recent trial results as well as what’s happening in terms of access in Europe and worldwide.
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.