Long-term treatment with Spinraza (nusinersen) was associated with improved or stable motor function in patients across the spinal muscular atrophy (SMA) disease spectrum, according to an analysis of real-world registry data. “To date, this is the largest prospective study over the longest observational period of [Spinraza] therapy in…
Treatment with the gene therapy Zolgensma (onasemnogene abeparvovec) led to improved breathing and motor function for a young boy who was experiencing unstable spinal muscular atrophy (SMA) symptoms on Spinraza (nusinersen), according to a case report from Japan. A year after receiving Zolgensma at the age of…
A team of scientists in Iowa have created a super minigene — a small gene fragment used as a tool to study genetic manipulation — that recapitulates essential features of the natural SMN1 and SMN2 genes linked to spinal muscular atrophy (SMA) and its severity. One of the most…
I was apprehensive when I was wheeled into my neurologist’s clinic on Jan. 18. I had taken my last dose of Evrysdi (risdiplam), a life-changing oral treatment that can improve motor function in SMA patients, during the last week of September. That was four months ago. “Why…
Difficulties with adequate nutrition and feeding persist among children and adolescents with spinal muscular atrophy (SMA) types 2 and 3 being treated with Spinraza (nusinersen), a Norwegian study reports. Important nutrients, such as protein, fiber, vitamins, and minerals, often were consumed in lower-than-recommended amounts by these patients, while…
I used to have this bracelet I loved. My dad bought it for me on a whim on my 16th birthday. We were walking through a high-end department store where he spotted a Swarovski counter. He immediately went up to the salesperson and said, “I want to find something for…
Children with spinal muscular atrophy (SMA), even those with substantial physical disability, tend to report that their quality of life is similar to other children their age, a small study from Germany reports. “The results from this pilot study show that German children with SMA, despite significant physical disability,…
Voyager Therapeutics is collaborating with Novartis to discover and develop next-generation gene therapies for conditions like spinal muscular atrophy (SMA). Under terms of the agreement, Novartis will obtain a target-exclusive license to Voyager’s TRACER capsids for SMA and Huntington’s disease, and it will…
Throughout 2023, SMA News Today brought you the most up-to-date news on scientific breakthroughs and treatment advances related to spinal muscular atrophy (SMA). These are the top 10 most-read articles we published throughout that year, with a brief reminder of what made them relevant to the SMA community. We…
Secretly, I usually dread this time of year. To borrow from singer-songwriter Sara Bareilles, December is, to me, a “problem child.” It’s not easy to exist inside of this contradictory month, a time of both celebration and solemnity, of looking backward and forward. My birthday and…