Author Archives: Steve Bryson PhD

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…

Prenatal Blood Test Accurately Gauges SMA Risk

UNITY Screen, a noninvasive prenatal blood test, can provide an accurate personalized risk assessment for hereditary diseases such as spinal muscular atrophy (SMA) in fetuses, according to a recent study. Offered by BillionToOne in the U.S., the single-gene noninvasive prenatal test (sgNIPT) included in UNITY Screen…

Scoliosis Progression Detailed in Untreated SMA Type 2 Children

Severe scoliosis — an abnormal curvature of the spine — was significantly associated with older age and limited motor abilities in children with spinal muscular atrophy (SMA) type 2 who had not received disease-modifying therapies, a study showed. These findings establish characteristics of untreated scoliosis progression on SMA…

Long-term Spinraza Treatment Found to Help SMA Teens, Adults

Up to 4.5 years of Spinraza (nusinersen) treatment led to meaningful improvements in motor function in adolescents and adults with spinal muscular atrophy (SMA), according to a medical records analysis. “[Spinraza] was effective in long-term follow-up,” researchers wrote. Noting a dearth of data on Spinraza’s long-term effectiveness…