Cure Spinal Muscular Atrophy

Trial-related informational needs and decision-making in the SMA community after broad treatment access: results of the 2nd Cure SMA clinical trial experience survey

Abstract Background When clinical trials were the only avenue for people with spinal muscular atrophy (SMA) to access potentially effective disease-modifying therapies (DMTs), people often traveled long distances for trials and were willing to tolerate significant treatment-relat...