Author Archives: Steve Bryson PhD

A new and noninvasive type of antisense oligonucleotide (ASO) therapy effectively treated spinal muscular atrophy (SMA) in a mouse model with established symptoms, a study reported. Since symptomatic and late-onset SMA patients are the most difficult to treat, “our [therapy] should be considered a great improvement from a clinical…

Spinraza (nusinersen) improved fine manual dexterity — the ability to make coordinated finger movements to grasp and manipulate objects — in both hands of five children with spinal muscular atrophy (SMA) type 2 over 1.5 years of treatment, a case series study showed. This study “is the first…

Delayed Spinraza (nusinersen) treatment due to the COVID-19 pandemic did not directly result in a worsening of symptoms in children with spinal muscular atrophy (SMA), a small study suggests. Any worse motor function that occurred during this period was attributed to a lack of response to the therapy…

Genetic analysis of dried saliva spots identified people with spinal muscular atrophy (SMA), a study demonstrated and possibly for the first time. The scientists noted that this noninvasive and low-cost, alternate method of genetic screening may be suitable for programs held in schools or workplaces without the need for…

Tranexamic acid (TXA) effectively reduced blood loss by over 50% during surgery to correct for scoliosis caused by spinal muscular atrophy (SMA), a 20-year study demonstrated. The medication also lowered the volume of blood transfused during surgery by 60%, and reduced the amount of crystalloid solutions used to maintain…

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, “…

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…

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…

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…

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…