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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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Information and Support over Christmas / New Year

11 December 2018 / Posted in: Information, Support

We are sorry we are unable to stay open for phone or email enquiries during the Christmas period. Please find details here of other organisations that will be available for you during this time.

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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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AVXS-101 to receive expedited regulatory review in the US and Europe

06 December 2018 / Posted in: Treatments & Research

Novartis has announced that the US Food and Drug Administration (FDA) has accepted for review with Priority Status their new drug application for the experimental gene therapy for SMA, AVXS-101, which has now been renamed ZOLGENSMA.

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UK SMA Research Day 2019

06 December 2018 / Posted in: Treatments & Research

The UK SMA Research Consortium and SMA UK are inviting all SMA Researchers to the next UK SMA Research Day, which will be held January 23rd 2019, from 9am to 5pm, in the Keele Hall Salvin Room at Keele University.

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Biogen awarded the 2018 International Prix Galien as Best Biotechnology Product for Spinraza (nusinersen)

06 December 2018 / Posted in: Treatments & Research

While we still await a decision from NICE, this marks the seventh Prix Galien for Spinraza, following country recognitions in the U.S., Germany, Italy, Belgium-Luxembourg, the Netherlands and the U.K.

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Have you visited a genetic service within the NHS? Could you share your opinions for research?

05 December 2018 / Posted in: Treatments & Research

Researchers are conducting a study to explore people’s views about how patient data could be used as part of future genomic medicine services in the NHS.

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Our virtual Christmas Tree is now live Our virtual Christmas Tree is now live

05 December 2018 / Posted in: Help Us

Choose a bauble to hang on our SMA UK tree and leave a festive message

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