Drug Treatments & Screening - What's Happening Now?
Find out more about this first approved treatment for SMA, how it works, clinical trials, and other studies and access plans for the UK.
On 24th May, the FDA in the USA announced their approval of this treatment. This one-time injection into the blood is designed to address the genetic root cause of SMA by replacing the faulty or missing SMN1 gene to limit the progression of the condition.
Track the development of possible SMA treatments in our Drug Pipeline and learn more about some of the drugs currently being tested.
This information sheet tells you more about how trials work, what to ask, and who to speak to if you are interested in joining one.
The appraisal process for nusinersen has clearly been shown to be ‘not fit for purpose’ for rare diseases treatments. Find out what work is being done to challenge this.
Programmes are beginning to be trialled in other parts of the world. In the UK, the National Screening Committee reviews whether such a programme should happen here. We submitted our views in September 2018 and are working with Genetic Alliance on a Patient Charter for access.