Every now and then, memories from my childhood hit me in the most unexpected ways, which is surprising because I don’t remember much from years past. Alas, my parents were cleaning out our basement recently when they came across a large trash bag filled with some of my stuffed animals.
Read more
Apitegromab, a muscle-strengthening therapy up for approval in the U.S., led to gains in motor function in a clinical trial for people with spinal muscular atrophy (SMA) who took disease-modifying therapies. Data from the trial, called SAPPHIRE (NCT05156320), formed the basis of Scholar Rock‘s recent…
Memories are like windows that look inward. Living with spinal muscular atrophy (SMA) certainly adds a special framework to that window, allowing light to pour in on all the unique experiences I wouldn’t have otherwise had. I consider that a blessing. SMA has taught me to treasure my…
After five years of treatment with Evrysdi (risdiplam), people with spinal muscular atrophy (SMA) types 2 and 3 continue to see stabilization and improvement in measures of motor function. That’s according to new long-term data from the Phase 2/3 clinical trial SUNFISH (NCT02908685), sponsored by Roche, which…
Second in a series. Read part one. My mum likes to say that my home-care nurse is like a second mother to me, and I’d have to agree. Ever since my SMA care was transferred from a pediatric to a general hospital after I turned 18, my home-care…
Children with spinal muscular atrophy (SMA) given the gene therapy Zolgensma (onasemnogene abeparvovec-xioi) in infancy are maintaining motor milestones after up to a decade of follow-up, according to new long-term clinical trial data. “At their last assessment, … patients [given the now-approved dosage of Zolgensma] were able to…
Get regular updates to your inbox.
