Most children with spinal muscular atrophy (SMA) treated with Spinraza (nusinersen) in a real-world setting had normal lab…
Articles by Steve Bryson, PhD
DNA contamination from sample processing remains a major problem in spinal muscular atrophy (SMA) newborn screening, a study reported.
Following treatment with gene therapy Zolgensma (onasemnogene abeparvovec-xioi), adding a second therapy, such as Spinraza (nusinersen) or…
Treatment with Spinraza (nusinersen) improved motor function in children, adolescents, and adults with spinal muscle atrophy (SMA) who…
Chronic pain is common among people with spinal muscular atrophy (SMA) across all age groups, according to a Swiss…
A two-week course of transcutaneous spinal cord stimulation (tSCS), a noninvasive method of stimulating the spinal cord using electrical impulses,…
SMN-K186R, a modified version of the SMN protein that is deficient in people with spinal muscular atrophy (SMA), was…
A program designed to provide data and tools for people with spinal muscular atrophy (SMA) identified through newborn screening…
A girl with spinal muscular atrophy (SMA) undergoing surgery to correct scoliosis, an abnormal sideways curvature of the…
A harness system designed for children with movement difficulties, the Portable Mobility Aid for Children (PUMA) improved or…