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Latest News: Research

FDA Accept Nusinersen For Priority Review

31 October 2016 / Posted in: Research

Biogen have announced that their New Drug Application (NDA) for Nusinersen has been accepted by the US Food and Drug Administration (FDA) for Priority Review.

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Biogen And Ionis Provide Update On European Nusinersen Approval Process

11 October 2016 / Posted in: Research

In response to requests for information, Biogen and Ionis provide an update on the progress made in moving the nusinersen development program forward.

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Views Sought On The Use Of Non-Invasive Prenatal Testing (NIPT)

06 October 2016 / Posted in: Research

The Nuffield Council on Bioethics is thinking about the ethical issues raised by NIPT and would like to talk to people over 18 who have SMA about their views on current and future uses of NIPT.

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Biogen and Ionis update on New Drug Application for Nusinersen

27 September 2016 / Posted in: Research

In response to requests for information, this update from Biogen and Ionis is for all in the SMA community.

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Roche Initiate Phase II Trial of RG7916

27 September 2016 / Posted in: Research

Roche have announced that they are to begin recruiting SMA patients in two parallel Phase II clinical trials of their second splice-modifying drug, RG7916 (also known as RO7034067).

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Molecular “Homing Devices” Drastically Improve SMA Therapy

22 September 2016 / Posted in: Research

Antisense oligonucleotide technology holds great promise for the treatment of SMA.

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Cure SMA Conference 2016 Report

20 September 2016 / Posted in: Research

Dr Hassan Sobati, SMA Support UK Trustee and parent, shares his experiences of attending this US event.

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ENDEAR clinical trial update

16 September 2016 / Posted in: Information & Support, Research

Principle Investigators in London and Newcastle have released this statement.

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The Basic Cellular Process Of Endocytosis Is Impaired In SMA Models

13 September 2016 / Posted in: Research

Thanks to two recently published scientific studies, we can now add endocytosis to the ever-growing list of pathways that the SMN protein is involved in.

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Avexis Provides Update on Ongoing AVXS-101 Trial

18 August 2016 / Posted in: Research

Avexis has released new information on their ongoing Phase I clinical trial of their gene therapy drug for SMA, AVXS-101.

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