Drug Treatments & Screening -
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.

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 26th February, the EMA CHMP recommended the approval of risdiplam for the treatment of 5q SMA in patients 2 months and older, with a clinical diagnosis of SMA Type 1, 2 or 3, or with 1-4 SMN2 copies. NICE has started its appraisal process and is currently due to publish a decision about access in July 2021.

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.

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.

Access Routes In The UK

Access Routes In The UK

This article outlines the processes that pharmaceutical companies go through in the UK to get a drug prescribed for an individual in their local pharmacy or hospital.

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.

Information For People Living Outside the UK

Information For People Living Outside the UK

As a small UK charity we regret we don't have the capacity to respond personally to individual enquiries when the person who has SMA is not resident in the UK. We hope the information on this page may answer some of your questions and be helpful to you.

NICE Systems Reviews

NICE Systems Reviews

There are many strands of work going on. You can read about them all and what we have done so far, here.

Research Into Treatments

Research Into Treatments

We outline five different approaches that have reached human clinical trial stages and show promise in treating SMA - bearing in mind that approximately 9 out of 10 experimental drugs starting clinical trials never gain final approval.