Author Archives: Steve Bryson PhD

The Canadian Agency for Drugs and Technologies in Health (CADTH) will not recommend reimbursement for Zolgensma in children with spinal muscular atrophy (SMA) who are more than 6 months old, citing insufficient evidence of efficacy and safety in older patients. “Other countries are providing guidance and funding based…

Children with spinal muscular atrophy (SMA) type 2 or 3 in China have lower bone mineral density — putting them at increased risk for bone fractures — compared with healthy children of the same age and sex, a study has found. Furthermore, SMA type 2 children had significantly…

Lung function and upper limb abilities worsened over a two-year period in young people with spinal muscular atrophy (SMA) type 3 who were unable to walk (non-ambulatory), a U.K. study has found. At the time of the study, the approved, disease-modifying therapy Spinraza (nusinersen) was unavailable to SMA…

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,”…

The gene therapy Zolgensma safely improved mobility in young children with spinal muscular atrophy (SMA) living in Qatar, a real-world study shows. The case series study, “Gene therapy for spinal muscular atrophy: the Qatari experience,” was published in the journal Nature Gene Therapy. Zolgensma…

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…

Many U.S. pediatricians fail to use developmental screening tools and are not familiar with the diagnostic requirements for spinal muscular atrophy (SMA), a study drawing on results of two surveys conducted by Cure SMA reported. These findings, from responses given by hundreds of pediatricians, support a continuing need to…

New data about Zolgensma, the one-time gene therapy for children with spinal muscular atrophy (SMA), demonstrate age-appropriate development when used pre-symptomatically, and rapid, meaningful efficacy in symptomatic children, even those with severe disease before treatment, according to two Phase 3 clinical trials. The therapy, developed by Novartis, uses…

Novartis will initiate SMART, a Phase 3b clinical study to evaluate the safety and efficacy of the one-time gene therapy Zolgensma (onasemnogene abeparvovec) in young children with spinal muscular atrophy (SMA) who weigh 8.5 to…

Poorer oxygen update in muscle cells and greater fatigue was evident among ambulatory spinal muscular atrophy (SMA) patients relative to healthy people in an exercise-based study, suggesting that problems in cell energy production play a role in this disease. A better understanding of…