The ICHOM Spinal Muscular Atrophy (SMA) project officially launched on January 8th, bringing together an engaged and highly committed Working Group of 34 clinicians, researchers, and patient representatives from across the globe. Participants span a wide range of geographies, including the Netherlands, Bulgaria, Brazil, Greece, Italy, Australia, South Africa, Malaysia, the Philippines, Türkiye, the United States, the United Kingdom, Saudi Arabia, India, and Canada – demonstrating both the global impact of SMA and the international commitment to improving outcomes for people living with the condition.

This project represents an important milestone for ICHOM, as it is the first time an ICHOM Set has been co-chaired by a patient representative. The Working Group brings together clinical expertise alongside lived experience, ensuring that the outcomes selected capture what truly matters to patients and families affected by SMA, while remaining robust, measurable, and applicable across healthcare systems.

Speaking after the kick-off call, Portia Thorman (SMA UK), the SMA patient co-chair, shared:

“Co-chairing the first SMA Project Working Group was a true honor. It highlighted just how much we can achieve through international teamwork when searching for reliable ways to measure patient outcomes. Most importantly, as a mum to a young man with SMA, witnessing the passion of clinicians from around the world and their eagerness to listen to our lived experiences gives me incredible hope for what the future holds for my son.”

The strong engagement and collaborative spirit evident from the outset underline the value of partnership between patients and professionals, laying a powerful foundation for the development of a globally relevant SMA ICHOM Set.