Therapy & care

SMA Europe aims to accelerate progress in the diagnosis, treatment and care of people living with SMA.

We do this through a range of activities:

Patient Advisory Groups (PAGs) – where we work closely with the pharmaceutical industries that have programmes for SMA. These PAGs are systematic, efficient and accountable groups of advocates who speak from the unique perspective of the SMA patient community. It advocates for the best possible research and equal access to the most innovative treatments.

Clinical Trial Readiness Project – initiated in 2018 and in collaboration with Cure SMA and the Industry Consortium in the US, this initiative seeks to understand and expand Europe’s current capacity to run clinical trials for potential SMA therapies.

Initiatives have included:

Network with Registries – Treat-NMD, RESTORE & PROMs