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...
SBMA Seen to Affect Skeletal Muscles First, Not Motor Neurons

SBMA Seen to Affect Skeletal Muscles First, Not Motor Neurons

Although a disease of motor neurons, nerve cells of the spinal cord and brainstem, the earliest signs of spinal and bulbar muscular atrophy (SBMA) are in skeletal muscles, according to data from a mouse study. These findings suggest that changes in muscle strength and...
Motor Gains and Safety with Zolgensma’s IT Use Continue, STRONG Data Show

Motor Gains and Safety with Zolgensma’s IT Use Continue, STRONG Data Show

Young children given a single injection of Zolgensma into the spinal canal continue to show gains in motor function and safety, new interim data from the STRONG trial in spinal muscular atrophy (SMA) type 2 patients up to age 5 show. These findings from the open-label...