Author Archives: Marisa Wexler MS

Even when given to young children with spinal muscular atrophy (SMA) at ages older than 6 months, the gene therapy Zolgensma (onasemnogene abeparvovec) can improve motor function, an analysis of real-world data show. Indeed, more than 70% of babies treated between 6 and 24 months had better scores…

Treatment with Evrysdi (risdiplam) continues to be safe and well-tolerated in people with spinal muscular atrophy (SMA) who have previously received other therapies for SMA, according to one-year data from the JEWELFISH clinical trial. Results also suggest that motor function has been stable in JEWELFISH participants after a year…

Treatment with Evrysdi (risdiplam) continues to be safe and well tolerated in people with spinal muscular atrophy (SMA) who have previously received other therapies for SMA, according to one-year data from the JEWELFISH clinical trial. Results also suggest that motor function has been stable in JEWELFISH participants after a…

Children and young adults with spinal muscular atrophy (SMA) types 2 and 3 continue to report improved or stabilized motor function after three years of treatment with oral Evrysdi (risdiplam) in the SUNFISH clinical trial. “The positive long-term efficacy and safety results for Evrysdi in this broad SMA population…

Most children with spinal muscular atrophy (SMA) who were presymptomatic and treated with Spinraza (nusinersen) in the NURTURE clinical trial are hitting major motor milestones within developmentally normal windows, new data show. “It’s only getting better. It’s spectacular,” Thomas Crawford, MD, from Johns Hopkins Medical Institute, said in…

Babies born in the Canadian province of Saskatchewan will now be tested for spinal muscular atrophy (SMA) as part of routine newborn screening. SMA is one of four medical conditions that have been added to the screening program administered to newly born infants by the Saskatchewan Health Authority. “Our…

A man with spinal muscular atrophy (SMA) type 2 in Japan who lived until his 60s was found after his death to have minimal damage in the brain but marked atrophy among the nerve cells of the spinal cord, an autopsy case report showed. “This report presents unique neuropathological…

Many prenatal genetic counselors are unfamiliar with important concepts related to spinal muscular atrophy (SMA) and its treatment, according to a new study. The study, “Evolving approaches to prenatal genetic counseling for Spinal Muscular Atrophy in the new treatment era,” was published in the Journal of…

The U.S. Food and Drug Administration (FDA) is giving priority review to a request that the oral therapy Evrysdi (risdiplam) be approved to treat pre-symptomatic infants with spinal muscular atrophy (SMA) who are less than 2 months old. Priority review is given to therapies with the potential to substantially…

Within the U.S., there is marked state-to-state variation in guidelines for whether Medicaid will cover Spinraza (nusinersen) or Zolgensma (onasemnogene abeparvovec) treatments, according to a new study. This finding raises concerns about equitable access to these lifesaving therapies for spinal muscular atrophy (SMA), according to researchers. The…