A new respiratory scoring system for evaluation of respiratory outcomes in children with spinal muscular atrophy type1 (SMA1) on SMN enhancing drugs
Lisa Edel, Christopher Grime, Victoria M. Robinson, A. Manzur, François Abel, Pinki Munot, Deborah Ridout, Mariacristina Scoto, Francesco Muntoni, Elaine Chan
Topics & Concepts
MedicineCohortRespiratory systemSpinal muscular atrophyInternal medicinePediatricsDiseaseNeurogenetic and Muscular Disorders ResearchMechanical Circulatory Support DevicesCongenital Anomalies and Fetal Surgery