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Latest News: Treatments & Research

NICE Announcement on Nusinersen

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.

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What Are Your Views On Screening Newborns For SMA?

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.

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Biogen Community Updates re Hydrocephalus

02 August 2018 / Posted in: Treatments & Research

We have received this community update which includes Q and As about the potential risk of hydrocephalus.

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Roche Community Update Re Risdiplam Development Programme

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.

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EMPoWER: Early Mobility and Powered Wheelchair Evidence Review

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

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Map of progress towards global access to nusinersen

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.

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Roche’s Sunfish Clinical Trial – why didn’t it happen here/what about access to future trials?

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.

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Consultation About Screening For SMA

27 June 2018 / Posted in: Treatments & Research

The National Screening Committee (NSC) has invited us to give comments and feedback on its June 2018 evidence review. We will be collating views on this.

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NICE Committee Meeting

27 June 2018 / Posted in: Treatments & Research

Today, we are at the NICE Committee Meeting in Manchester which is considering whether to recommend access to nusinersen; we will know the outcome in 3 – 4 weeks’ time. Thanks to the other two Patient Representatives who are also attending.

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PTC Therapeutics Present Interim Data from FIREFISH Clinical Trial

21 June 2018 / Posted in: Treatments & Research

PTC Therapeutics recently presented preliminary data from the FIREFISH Phase II clinical trial of their splice-modifying drug, RG7916, at the 2018 Cure SMA conference in Dallas, USA.

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