Juliana F. Mazzeu

Gene therapy benefits in a small cohort of symptomatic 5q spinal muscular atrophy patients and real world pediatric outcomes

OBJECTIVE: To characterize clinical outcomes of gene replacement therapy administered after symptom onset in a small group of children with 5q spinal muscular atrophy (SMA) and to describe its impact on motor function and supportive care needs in real-world practice. METHODS: Obs...