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 19th May 2020, the European Commission approved IV-delivered Zolgensma® for the treatment of specific groups of babies and young children. In England, NICE is progressing its appraisal as to whether to recommend the treatment for NHS funding, as is the SMC in Scotland. Decisions are anticipated in March / April 2021.
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.
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.
In July 2019, NICE confirmed details of its plans to review the case for change to the methods it uses to develop guidance on drugs, medical devices and diagnostics. This page shows the timeline for the next stages of the review.
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.