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.
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.
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.
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.
Find out more about: drugs currently being tested in clinical trials; information on how clinical trials work; more about the NICE process.
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.
Some Q&As for things to think about when considering accessing treatments, for example overseas.
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.
Our Research Correspondents report on the available, but limited, research on rarer forms of 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.