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 24th May, the FDA in the USA announced their approval of this treatment for children under age 2 years. Pharmaceutical company AveXis has filed for licensing in Europe. In England, the regulatory authority NICE has started its investigations.

Risdiplam / RG7916

Risdiplam / RG7916

On 14th January 2020, Roche announced a limited pre-approval global access programme for this orally-administered liquid. A US licensing decision is expected in May 2020. Roche anticipate filing for authorisation in Europe in mid-2020.

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.

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.

Global Access Concerns

Global Access Concerns

Some Q&As for things to think about when considering accessing treatments, for example overseas.

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.

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.