Columns

Children’s Breathing Problems at Night Tied to Zolgensma Use

Despite its well-reported benefits in motor function, the gene therapy Zolgensma (onasemnogene abeparvovec-xioi) may not be as effective at preventing or easing breathing problems at night in young children with spinal muscular atrophy (SMA), according to a small real-world study. Nighttime breathing problems were found to occur in…

What if I Could Take a Vacation From SMA?

“Yesterday, All my troubles seemed so far away, Now it looks as though they’re here to stay, Oh, I believe in yesterday.” Among the countless hits by the Beatles, “Yesterday,” released in 1965, has stood the test of time as one of the most revered and influential songs in…