Nusinersen (SpinrazaTM)

Pages in this website section last updated 23rd November 

Recent Headlines

23rd Novemeber 2020
SMA UK Sends Evidence to Biogen for the NICE Review Considering Whether People who have SMA Type 3 and Unable to Walk can Benefit from Nusinersen Treatment

20th November 2020
If you or your child live in England, have SMA Type 3 and have lost the ability to walk, please respond to this survey

29th October 2020
NICE Announces Start of Review to Consider if People who have SMA Type 3 who're Unable to Walk can Benefit from Nusinersen Treatment

Scroll down further for:

  • What is it?
  • How it works
  • UK Access Now
  • Treatment Information Leaflets
  • Clinical Trials and 'Real World' Use
  • Global Access
  • The Road to UK Access: Autumn 2016 – July 2019

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What is it?

In collaboration with researchers, nusinersen was developed by Ionis Pharmaceuticals and Biogen Idec. Marketed as SpinrazaTM, it is the first (and currently, the only) potentially disease-modifying treatment available in the UK for 5q SMA, which includes SMA Type 1, 2 3 and 4. It is an antisense oligonucleotide designed to modify the product of the SMN2 “backup” gene to produce more functional SMN protein.

You can read how the original scientists who worked on it won the 2019 Breakthrough prize (the “Oscars for science”) here; how Biogen won the UK Prix Galien award for it, here.

How it Works

You can read more and watch Cold Spring Harbor laboratory’s video about how it works, here.

UK Access Now

The NHS now funds this treatment for children and adults but there are criteria which limit who has access.

Read more

Treatment Information Leaflets

These leaflets aim to provide a summary for anyone considering the possibility of treatment for their child, or themselves. Read More.

Clinical Trial Results and 'Real World' Use

Clinical trials have been with infants and children who have SMA Types 1, 2 or 3. There have not yet been any clinical 
trials with anyone with adult onset SMA Type 4.

Our Research Correspondents have tracked the results as they have been released. You can read these updates here. They also summarise published ‘real world’ studies, here

Global Access

On 23rd December 2016, the US Food and Drug Administration (FDA) approved the use of nusinersen under its brand name SpinrazaTM for both children and adults with SMA Types 1, 2 or 3 SMA.

On 1st June 2017, the European Commission approved the treatment for those with 5q SMA. Their full report is here. Since then, access in each country has been, and still is being, decided by their regulatory authorities.

You can find out what progress countries have made here.

The Road to UK Access: Autumn 2016 – July 2019

You can read about this long and stressful journey for the SMA Community, here

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