Coffee and Cake Day Coffee and Cake Day

04 January 2019 / Posted in: Help Us

Beat the Winter Blues by getting friends and family together for some delicious bakes

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CQC Survey: People's Experiences of Care

04 January 2019 / Posted in: Information

The Care Quality Commission is running a survey to find out people's different experiences of care. By sharing your experiences, you’ll help them to improve the overall quality of care in England.

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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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AveXis Issues U.S. Community Statement on BLA Acceptance

12 December 2018 / Posted in: Treatments & Research

AveXis has provided the following U.S. community statement on the BLA submission and acceptance for their investigational therapy, AVXS-101 - now known as ZOLGENSMA.

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We are Supporting Genetic Alliance UK's ‘Resetting the Model’ Project

11 December 2018 / Posted in: Treatments & Research

We are taking every opportunity to attend APPGs to give voice to the SMA Community’s frustrations and distress caused by the NICE appraisal process for nusinersen. We are supporting this ambitious project aimed at improving the way that future decisions on rare disease medicines are made as a positive way forward.

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