Page last checked: 19th December 2021

Last updated: 15th February 2022

Individuals and their families should discuss appropriate treatment options with their clinical team in their specialist neuromuscular centre.

The most optimal treatment for any individual will be based on a number of medical considerations, and individual patients’ choice. All specialist neuromuscular centres will be able to treat eligible patients with risdiplam. If an individual is no longer in touch with a specialist neuromuscular centre, they should contact either the centre where they received care previously or their nearest centre. Individuals should discuss genetic testing with their clinical team.

By the end of November 2021, 215 eligible patients in England had already started treatment with risdiplam as part of the Early Access to Medicines Scheme (EAMS), which closed in July 2021. There remained a small number of patients who are eligible for treatment including those who:

• have been diagnosed since July 2021;
• are no longer able to be treated with nusinersen (Spinraza);
• have SMA Type 3 who were not eligible for the EAMS;
• are cared for by specialist neuromuscular centres that did not initiate individuals on treatment during the EAMS.

If you’re one of these people, you should contact your specialist neuromuscular centre in the first instance.