Author Archives: Marisa Wexler MS

Most of the children with spinal muscular atrophy (SMA) who received the gene therapy Zolgensma (onasemnogene abeparvovec) in their first weeks of life are able to walk independently within developmentally normal age ranges, according to new research from the SPR1NT clinical trial. The findings were presented at this year’s…

Most of the children with spinal muscular atrophy (SMA) who received the gene therapy Zolgensma (onasemnogene abeparvovec) in their first weeks of life are able to walk independently within developmentally normal age ranges, according to new research from the SPR1NT clinical trial. The findings were presented at this…

In the RAINBOWFISH trial testing oral Evrysdi (risdiplam) in babies with spinal muscular atrophy (SMA) but no evident symptoms, most of the seven children treated for at least one year were able to stand and walk within developmentally normal windows. These new data were presented at the Muscular Dystrophy Association…

In the RAINBOWFISH trial testing oral Evrysdi (risdiplam) in babies with spinal muscular atrophy (SMA) but no evident symptoms, most of the seven children treated for at least one year were able to stand and walk within developmentally normal windows. These new data were presented at the Muscular Dystrophy Association…

In the RAINBOWFISH trial testing oral Evrysdi (risdiplam) in babies with spinal muscular atrophy (SMA) but no evident symptoms, most of the seven children treated for at least one year were able to stand and walk within developmentally normal windows. These new data were presented at the Muscular Dystrophy…

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…