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.

Drugs Currently Being Tested In Clinical Trials

Drugs Currently Being Tested In Clinical Trials

Track the development of possible SMA treatments in our Drug Pipeline and learn more about some of the drugs currently being tested.

How Clinical Trials Work

How Clinical Trials Work

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 NICE Process - Work to change it and what happens in the rest of the UK

The NICE Process - Work to change it and what happens in the rest of the UK

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.

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.