Pages in this website section last updated 23rd July 2020
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
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.
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.
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.
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.