Position Statement: Sharing of Clinical Research Data in Spinal Muscular Atrophy to Accelerate Research and Improve Outcomes for Patients

Recent years have seen increasing clinical research activities in spinal muscular atrophy (SMA), involving patients, their families, clinicians, researchers, regulators and industry This has led to unprecedented advancements in understanding of genetic determinants of severity and prognosis, the natural history, outcome measures, and most importantly first marketed therapies However, many patients with SMA do not benefit yet from effective treatments or show limited clinical response or impact on their quality of life.