Real-World Treatment with Risdiplam in Adults with SMA

This technical report presents interim results from a multicenter study on the use of risdiplam in adults diagnosed with spinal muscular atrophy (SMA) who are 25 years of age or older. The study aims to enhance understanding of patient characteristics, treatment patterns, and clinical outcomes associated with risdiplam treatment. Data was collected from medical records of 20 patients across three sites, with a cutoff date of April 23, 2024. Key findings include the mean age at treatment initiation being 40.3 years, with a majority diagnosed with 5q SMA. The report outlines treatment data, including rehabilitation therapies utilized by patients during the follow-up period. It also notes that most patients were alive at the end of the follow-up, with varying durations of therapy and rehabilitation. The study addresses the critical need for descriptive information regarding the management and outcomes of adults with SMA receiving risdiplam, contributing to the existing literature on this patient population.