Spinraza-Zolgensma Combination Well-tolerated in Children with SMA Type 1, Study Shows

Spinraza-Zolgensma Combination Well-tolerated in Children with SMA Type 1, Study Shows

Combining Spinraza (nusinersen) with the gene therapy Zolgensma (onasemnogene abeparvovec-xioi) is generally well-tolerated and sustains motor improvements in children with spinal muscular atrophy (SMA) type 1, according to a case series study. The data, which...
Respiratory Weakness in SMA Most Pronounced in Childhood, Study Finds

Respiratory Weakness in SMA Most Pronounced in Childhood, Study Finds

Lung function in people with earlier-onset forms of spinal muscular atrophy (SMA) tends to decline gradually throughout childhood and stabilizes in early adulthood, according to data from a population-based natural history study. The study, “Natural history of lung...
Lung Ultrasound Reliable Option to Chest X-rays for Monitoring Children

Lung Ultrasound Reliable Option to Chest X-rays for Monitoring Children

Lung ultrasound is a safe and reliable bedside tool to detect atelectasis (partial lung collapse) early in children with neuromuscular diseases, including spinal muscular atrophy (SMA), a study suggests. This radiation-free method could be used to monitor lung health...