Author Archives: Steve Bryson PhD

New Spinraza Delivery Technique for Patients With Spinal Deformities

For spinal muscular atrophy (SMA) patients with spinal deformities, administration of Spinraza (nusinersen) by a novel subcutaneous intrathecal catheter system improved upper limb function, a small study reports. This delivery technique, however, increased the risk of mechanical malfunction and infections, the researchers noted. The study, “…

Anxiety, Depression a Problem for School-age Patients in China

School-age children and adolescents with spinal muscular atrophy (SMA) in China experience a high rate of anxiety and depression, a questionnaire-based study suggests. “Professional psychological care [may be] included in the standard of care,” the researchers wrote, noting that a higher prevalence of depression was seen among students with…

Cure SMA Advocates for Better Wheelchair Storage for Air Travel

In a letter to a U.S. Department of Transportation Advisory Committee, Cure SMA called for better wheelchair handling and storage for people with spinal muscular atrophy (SMA) who travel by air. The patient advocacy organization shared its perspective on the challenges of airplane travel — from booking tickets to…

Mothers Want to Improve Children’s Quality of Life

Mothers of children and adolescents with spinal muscular atrophy (SMA) want to find ways to improve the quality of life of their children and families, a questionnaire-based study reports. “The results of this research … could help specialists in providing psychological support for families of SMA children and/or adolescents,”…

Early Work Supports Apitegromab’s Safety as SMA Muscle Therapy

A series of toxicology studies in rats and macaque monkeys confirmed the safety and tolerability of apitegromab, an investigational spinal muscular atrophy (SMA) therapy, supporting its ongoing assessment in patient trials, a study reported. The study, “Preclinical Safety Assessment and Toxicokinetics of Apitegromab, an Antibody Targeting…