Drug Treatments -
What's Happening Now?

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

These pages tell you about any drug treatments that have been approved for use, any in the pipeline, and other related topics. Whatever the treatment, it remains vital that anyone who has SMA also has care and management as outlined in the internationally agreed Standards of Care for SMA.

Treatments Funded by the NHS in the UK

Treatments Funded by the NHS in the UK

The NHS in the UK now funds three potential treatments. Access to these depends on residency and any other conditions that affect a child, young person or adult’s rights to access specialised NHS treatment as well as meeting the eligibility criteria that apply to the specific treatment.

Spinraza / Nusinersen

Spinraza / Nusinersen

Pharmaceutical company Biogen's drug is the first internationally-approved treatment for SMA. There is now access in the UK, though this is not possible for all with SMA Type 1, 2 or 3.

Zolgensma / onasemnogene abeparvovec / AVXS-101

Zolgensma / onasemnogene abeparvovec / AVXS-101

On 19th May 2020, the European Commission approved IV-delivered Zolgensma® for the treatment of specific groups of babies and young children. On 8th March 2021, access agreements for potentially eligible children were announced for England and Scotland.

Risdiplam / Evrysdi / RG7916

Risdiplam / Evrysdi / RG7916

On 19th November 2021, NICE, the NHS and Roche agreed a 3-year Managed Access Agreement for Risdiplam for those who have SMA Type 1, 2 or 3. The Scottish Medicines Consortium is well underway with its review of whether to recommend access.

Trials Testing Combined Drug Treatments

Trials Testing Combined Drug Treatments

This is where we provide updates on any clinical trials and trial results exploring combining different drug treatments.

Other Potential Treatments

Other 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.

Our Work Advocating For Access

Our Work Advocating For Access

Since Autumn 2016, we have invested a huge amount of time and energy to advocate for access to all three treatments (nusinersen, zolgensma and risdiplam) in the UK, as well as joining with others to voice the need for reform of regulatory systems and processes.

Research & Treatment Approaches

Research & Treatment Approaches

Here, we provide information on basic research into SMA, different treatment approaches, and SMA biomarkers, as well as interviews with scientists working on SMA.

UK Residents Considering Applying To Join An Overseas Trial

UK Residents Considering Applying To Join An Overseas Trial

There are lots of things to think about if you're considering this. This page answers some of the questions you may have and gives further information.