On Tuesday 3rd February 2026, the Managed Access Agreement (MTA) committee met to consider evidence relating to Risdiplam and Spinraza for the treatment of spinal muscular atrophy (SMA). We would like to begin by sincerely thanking everyone who took the time to complete our recent gap-filling survey.

The survey results were instrumental in addressing several complex challenges faced by the committee, particularly in relation to the economic modelling of treatment. Your input helped clarify important considerations such as the appropriate number of carers to include in the model, as well as capturing meaningful treatment impacts where published data remain limited. These included reductions in hospital admissions and improvements in school attendance and participation in work.

Overall, the meeting felt constructive. As is often the case with rare conditions, there will never be the volume of data typically sought in more common diseases, and this reality was clearly acknowledged. Against this backdrop, the contributions from patient and clinical experts were especially valuable. They did an excellent job of articulating the wide-ranging benefits of treatment across the full SMA spectrum, highlighting impacts not only on physical health but also on independence, daily life, and family wellbeing.

We expect a decision from the committee in approximately one week. Until then, thank you once again to everyone who contributed their time and expertise. We will provide further updates as we hear.