Drug Treatments & Screening - What's Happening Now?

Although there’s currently no cure for SMA, this doesn’t mean that nothing can be done.

There are a range of options aimed at managing symptoms, reducing complications of muscle weakness and maintaining the best quality of life. These are outlined in the internationally agreed Standards of Care for SMA. There’s also a considerable amount of research into SMA taking place around the world, not only improving our understanding of the condition, but also helping to develop effective drug treatments. These pages tell you about any that have been approved for use, any in the pipeline, and other related topics.

Nusinersen

Nusinersen

Find out more about this first approved treatment for SMA, how it works, clinical trials, and other studies and access plans for the UK.

Zolgensma / AVXS-101

Zolgensma / AVXS-101

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.

Potential Treatments

Potential Treatments

Find out more about: drugs currently being tested in clinical trials; information on how clinical trials work; more about the NICE process.

Research into Rarer Forms of SMA

Research into Rarer Forms of SMA

Our Research Correspondents report on the available, but limited, research on rarer forms of SMA.

Newborn Screening for SMA

Newborn Screening for SMA

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.