Author Archives: Steve Bryson PhD

MicroRNAs in Blood May Predict Spinzara Response in Type 1 Patients

MicroRNAs isolated from the bloodstream of patients with infant-onset spinal muscular atrophy (SMA) type 1 before treatment were related to functional responses to Spinraza (nusinersen) after six months of therapy, a study suggested. Researchers said these microRNAs may be potential biomarkers to predict or monitor patients’ responses to…

Combining Evaluation Tools May Detect Early Signs of SMA: Study

Combining two clinical assessment tools helped recognize early neurological signs in infants with spinal muscular atrophy (SMA) who were identified through a newborn screening program, a study suggested. Even in the absence of obvious clinical signs, infants typically defined as presymptomatic may have minimal signs, such as poor muscle…

MicroRNAs Can Predict Response to Spinraza, Study Suggests

MicroRNAs isolated from people with spinal muscular atrophy (SMA) types 2 and 3 before treatment predicted eventual responses to Spinraza (nusinersen) therapy, a study suggested. The study, “Muscle microRNAs in the cerebrospinal fluid predict clinical response to nusinersen therapy in type II and type III…

Combining Therapies May Improve Efficacy in SMA: Mouse Model

A combined spinal muscular atrophy (SMA) therapy — one incorporating two treatments targeting different disease processes related to the SMN protein — showed improved efficacy over single treatments in a mouse model. The findings support further research into combination treatment strategies, the researchers said. A combined therapy could potentially…

Spinraza Most Effective in Younger Type 1 Children: Real-world Study

In a real-world study involving more than 40 Swiss patients with spinal muscular atrophy (SMA), Spinraza (nusinersen) was found to effectively improve motor function — particularly among younger children with SMA type 1. Older treated patients gained or stabilized their motor function, but infections prompted the need for ventilation and…