Like so many children affected by this ultra-rare disease, Salim and his family navigate the complexities of SMA-PME with remarkable strength and determination. SMA-PME is caused by mutations in the ASAH1 gene, which leads to a deficiency in acid ceramidase — an enzyme critical to cellular health. The disease progressively affects a child's ability to walk, speak, eat, and breathe.
For Salim and his family, every day is both a challenge and a reminder of why this research cannot wait. They are not alone: our community of families spans the globe, connected by a shared determination to find answers and to support one another through the unimaginable.
Salim's full story will be shared here with his family's permission. In the meantime, we invite you to learn about the disease, connect with our community, and support the research that could change everything for children like Salim.
