Spinraza helps SMA type 3 motor function, maybe not fatigability
Treatment with Spinraza (nusinersen) extended the distance adults with spinal muscular atrophy (SMA) type 3 could walk during…
Articles by Steve Bryson, PhD
Fatty liver disease may be common complication of SMA: Small study
An abnormal buildup of fat in the liver, referred to as fatty liver disease, was found to be common in…
Spinraza shows safety across real-world lab tests given young children
Most children with spinal muscular atrophy (SMA) treated with Spinraza (nusinersen) in a real-world setting had normal lab…
Study finds DNA contamination persists in SMA newborn screening
DNA contamination from sample processing remains a major problem in spinal muscular atrophy (SMA) newborn screening, a study reported.
No significant gains seen with SMA type 1 dual therapy in real world
Following treatment with gene therapy Zolgensma (onasemnogene abeparvovec-xioi), adding a second therapy, such as Spinraza (nusinersen) or…
Spinraza treatment improves motor function in nonambulatory SMA
Treatment with Spinraza (nusinersen) improved motor function in children, adolescents, and adults with spinal muscle atrophy (SMA) who…
Chronic pain is common in SMA across all age groups: Swiss study
Chronic pain is common among people with spinal muscular atrophy (SMA) across all age groups, according to a Swiss…
Noninvasive spinal cord stimulation may help SMA motor function
A two-week course of transcutaneous spinal cord stimulation (tSCS), a noninvasive method of stimulating the spinal cord using electrical impulses,…
Modified SMN protein aids gene therapy efficacy: Mouse study
SMN-K186R, a modified version of the SMN protein that is deficient in people with spinal muscular atrophy (SMA), was…
LTFU model helps SMA families, clinicians, NBS programs: Study
A program designed to provide data and tools for people with spinal muscular atrophy (SMA) identified through newborn screening…