Author Archives: Marisa Wexler MS

People with spinal muscular atrophy (SMA) types 2 or 3 who start treatment with Spinraza (nusinersen) and show less muscle damage on MRI scans are more likely to see motor function improvements, a new study reports. The finding “underscores the importance of early diagnosis and treatment to…

Without disease-modifying treatment (DMT), spinal muscular atrophy (SMA) carries high mortality rates and steep healthcare costs, a study in Hong Kong reported. Not surprisingly, the greatest loss of life and highest costs were observed in children with SMA type 1, one of this disease’s most severe forms…

A new system that contains a gene-modulating therapy attached to an antibody that enables crossing into the nervous system improved survival outcomes in a mouse model of severe spinal muscular atrophy (SMA), a new study reports. The study, “Antibody-oligonucleotide conjugate achieves central nervous system delivery…

Children with spinal muscular atrophy (SMA) who have lower levels of an inflammatory protein called interleukin-8 (IL-8) in the fluid surrounding the brain and spinal cord tend to respond better to treatment with Spinraza (nusinersen), a new study suggests. The findings indicate that IL-8 is a better predictor…

After two years of treatment with Evrysdi (risdiplam), most babies with spinal muscular atrophy (SMA) type 1 in the FIREFISH clinical trial are still alive without a need for permanent ventilation, and many of the youngsters are showing continual improvements in motor development. The results were published…

Continued treatment with apitegromab, designed to strengthen muscles, led to stabilization or improvement in measures of fatigue and life quality for children and young adults with types 2 and 3 spinal muscular atrophy (SMA), according to new data from the TOPAZ clinical trial. According to apitegromab’s developer…

Children with spinal muscular atrophy (SMA) switching from Spinraza (nusinersen) to Evrysdi (risdiplam) are generally optimistic about the switch as are their caregivers, according to a new study. The report, “How children and caregivers viewed the change from nusinersen to risdiplam for treating…

Levels of an immune protein called CHIT1 change over time in children with spinal muscular atrophy (SMA) who undergo treatment with Spinraza (nusinersen), a small study reports. The findings thus suggest that the protein’s levels — in the cerebrospinal fluid or CSF, the liquid surrounding the brain…

Treatment with Evrysdi (risdiplam) increased levels of SMN protein and stabilized motor function in a Phase 2 clinical trial that enrolled spinal muscular atrophy (SMA) patients who had previously been on other therapies, two-year data show. “These important data demonstrate the safety and efficacy of Evrysdi in…

The gene therapy Zolgensma (onasemnogene abeparvovec-xioi), approved in the U.S. for very young children with spinal muscular atrophy (SMA), potentially could work for adults with the rare genetic disorder, a new study suggests. The study reports that few adults with SMA were found to have antibodies against…