Latest News: Treatments & Research
Biogen Announces November Closure of the Expanded Access Programme for Infants Newly Diagnosed with SMA Type 1
23 August 2018 / Posted in: Treatments & Research
We urged Biogen not to take this step which creates yet more immense distress and worry for families. We will continue to focus our advocacy for access to treatment on the urgent need for NICE, NHS England and Biogen to agree the terms of a managed access agreement to replace this EAP ahead of 1 November, as well as the need for a long-term sustainable plan for access.
21 August 2018 / Posted in: Treatments & Research
Clinicians and SMA Charities yesterday expressed the need for urgent action. You can read the letter sent to NICE, NHS England and the National Institute for Health Research, here.
15 August 2018 / Posted in: Treatments & Research
Biogen has provided this community statement in response to yesterday's announcement from NICE.
13 August 2018 / Posted in: Treatments & Research
Nusinersen not recommended for funding by NICE is the devastating decision for so many, announced today. One glimmer of hope, however, is that it does leave the door open for a Managed Access Agreement. Comments are needed by 5th September.
03 August 2018 / Posted in: Treatments & Research
The National Screening Committee (NSC) has invited us to give comments and feedback on its June 2018 evidence review. Their current recommendation is ‘systematic population screening programme not recommended’. When we submit our response, we would like to represent views from the SMA community about screening newborns for SMA. You can let us know your thoughts and take our survey.
02 August 2018 / Posted in: Treatments & Research
We have received this community update which includes Q and As about the potential risk of hydrocephalus.
24 July 2018 / Posted in: Treatments & Research
During the recent Cure SMA researcher meeting in USA, Roche shared progress details of the risdiplam programme which you can read more about here. The update also includes an acknowledgement of the disappointment that UK SUNFISH trial sites were not initiated in time to allow UK patients to take part.
05 July 2018 / Posted in: Information, Treatments & Research
We are pleased to be involved with a new project to estimate the costs and benefits of providing young disabled children under 5 years with powered mobility aids
05 July 2018 / Posted in: Treatments & Research
We have received an updated map from Biogen which shows the latest status of global access to nusinersen.
28 June 2018 / Posted in: Treatments & Research
We asked GOSH if they could throw any light on this and what assurances they could give that the UK won’t miss out in the future. Also, we checked what actions anyone interested in being considered for a clinical trial should take.