Latest News: Treatments & Research

National programme for carrier or newborn screening for SMA not recommended by UK National Screening Committee (NSC)

18 February 2019 / Posted in: Treatments & Research

This was their conclusion following their consultation and evidence review. We will continue to work with other groups to push for both a review of the evidence criteria and a further urgent review of a newborn screening programme for 5q SMA. You can read our submission to the consultation and more about NSC's recommendation here.

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Please invite your MP to the 25th February Parliamentary ‘drop in’ about access to nusinersen (Spinraza) for England, Wales and Northern Ireland

14 February 2019 / Posted in: Treatments & Research

MDUK and SMA UK have jointly arranged this event. Parents and adults representing the SMA Community will be telling MPs about their experiences of living with SMA, and will be joined by clinicians to talk about the importance of access to nusinersen. They will be outlining the actions MPs can take to show their support and raise the matter further.

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Subject to successful sign off, nusinersen is expected to be routinely available in Scotland for those with SMA Types 1, 2 and 3 from April 2019

12 February 2019 / Posted in: Treatments & Research

Nusinersen has been routinely available to children with SMA Type 1 in Scotland since May 2018. This will continue. The SMC has now granted nusinersen ultra-orphan designation, allowing the treatment to be reviewed for those with SMA Types 2 and 3 through its new ultra-orphan appraisal process with the prospect of it being made available for at least three years while further outcome data is gathered.

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NICE announce Committee Meeting on 6th March 2019

01 February 2019 / Posted in: Treatments & Research

This will be the third meeting to review whether NICE will recommend nusinersen for funding by NHS England. This meeting will be in Manchester again. As soon as we have more details we will let you know.

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Urgent request: short survey to assist with ongoing negotiations with NICE re access to nusinersen

17 December 2018 / Posted in: Treatments & Research

We are facilitating finding out more about the financial and work-related impact of living with SMA Type 1, 2, 3 or 4, for unpaid carers in the UK. This survey may be completed by the person with SMA, partners, parents / carers, or together. Closing date for the survey is 9am on Monday 7th January 2019.

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Roche's Risdiplam Granted PRIME Designation by EMA

17 December 2018 / Posted in: Treatments & Research

Roche have issued a media release confirming PRIME designation has been granted by the European Medicines Agency (EMA) for risdiplam (RG7916) for the treatment of SMA.

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Response from Biogen to Charities' Letter re Bridging Solution

14 December 2018 / Posted in: Treatments & Research

We have received a written response from Biogen to the joint charities' letter we sent on 29th November about the urgent need to set up a bridging solution for access to nusinersen.

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Response from NICE to Charities' Two Joint Letters

14 December 2018 / Posted in: Treatments & Research

NICE have sent a letter back in response to the joint charities' two letters we sent: firstly about the urgent need to set up a bridging solution for access to nusinersen; secondly urging them to ensure nusinersen has priority place at the next meeting.

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Call for Expressions of Interest: Patient Representatives wanted to participate in PARADIGM Project

13 December 2018 / Posted in: Treatments & Research

PARADIGM's objective is to develop processes and tools to guarantee patient engagement in three key decision-making points in medicines research and development. Closing date for applications is 28th December.

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Biogen Issues Global Community Statement on Spinraza

13 December 2018 / Posted in: Treatments & Research

Biogen has provided a global community statement on Spinraza. The statement covers worldwide statistics, a summary of data as presented at the 23rd International Congress of the World Muscle Society, and plans to "broaden their development portfolio".

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