Author Archives: Marisa Wexler MS

#MDA2022 – Newborns Given Evrysdi Standing, Walking Year Later

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…

#MDA 2022 – Evrysdi Safe at 1 Year in Previously-treated Patients

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…

Newborn Screening for SMA Now Launched in Canadian Province

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…