P233 Zolgensma in spinal muscular atrophy: a Toronto paediatric hospital experience

Spinal muscular atrophy (SMA) is a neuromuscular disorder that is characterized by loss of motor neurons leading to progressive muscle weakness and atrophy. SMA is a lifelong disorder that impacts a person's quality of life and leads to significant morbidity and mortality. Since the addition of SMA on Newborn Screening programs, healthcare professionals have been able to intervene and provide therapies to infants prior to symptom onset of SMA. A favourable approach to therapy is Zolgensma, a one-time gene therapy approved for the treatment of SMA in Ontario to newborns who are 6 months of age or less.
Source: Neuromuscular Disorders - Category: Neurology Authors: Source Type: research