SMA treatment Zolgensma seen to benefit patients in real world
Sep 13, 2024 · Treatment with Zolgensma (onasemnogene abeparvovec-xioi) improves motor function in children with spinal muscular atrophy (SMA) who carry at least four copies of the so-called backup SMN2 gene, according to real-world data from the RESTORE patient registry. SMA children treated with the approved gene therapy achieved several motor milestones at ...
HCP SMA News Today – The Web's Leading Resource For People …
Oct 10, 2018 · Surgery to correct scoliosis, a sideways curvature of the spine, slowed lung function decline in people with spinal muscular atrophy (SMA) type 2 and 3 who had yet to receive treatment, a study focusing on secondary outcomes reported. While all participants reported overall satisfaction with posture and…. September 16, 2024 News by Steve ...
Spinal Muscular Atrophy: Cardiac Disease – HCP SMA News Today
Spinal Muscular Atrophy (SMA) is an inherited neurodegenerative disease, 1 but it is increasingly clear that the pathology of SMA extends beyond ventral horn spinal cord neurons and includes the heart among other organs. 2 In patients with SMA, tissue levels of SMN1 protein are reduced in heart tissues, 3 and numerous mouse models of SMA exhibit cardiac developmental abnormalities which ...
How the pandemic changed online school for the better
Sep 16, 2024 · Online school was cutting-edge at the time; it wasn’t hailed as a viable alternative to in-person classes as it is today. The platforms were clunky and, at times, trivialized. Professors were slow to adapt, and often reluctant. As perpetually online as I was, I made the transition with ease, but I can see how students with less experience ...
Apitegromab still aiding SMA patients in TOPAZ, Phase 3 data on …
Aug 14, 2024 · Apitegromab aims to promote better strength, mobility in SMA patients. The Phase 3 SAPPHIRE trial (NCT05156320) has enrolled 188 children and young adults with SMA type 2 or type 3, all without the ability to walk but able to sit independently. Patients are being given monthly infusions of apitegromab or a placebo for one year, on top of ...
Real-world benefits of Zolgensma for SMA seen in RESTORE data
Jan 31, 2024 · RESTORE (NCT04174157) is a real-world, observational patient registry that’s tracking outcomes for up to 15 years and seeks to capture responses to DMTs that weren’t available in earlier registries. Here, the scientists described clinical outcomes for 168 registry participants who’d been treated with Zolgensma as of May 2022 and hadn’t received any prior SMA therapies.
Spinal Muscular Atrophy: Nutrition and Supplementation
The nutritional management of patients with progressive neuromuscular diseases like Spinal Muscular Atrophy (SMA) is complex and is achieved optimally with an individualized approach from a multidisciplinary care team. 1,2 Patients with SMA need nutritional monitoring at least annually with assessment of caloric intake and standard ...
Surgery for scoliosis slows SMA lung function decline: Study
Sep 18, 2024 · Surgery to correct scoliosis, a sideways curvature of the spine, slowed lung function decline in people with spinal muscular atrophy (SMA) type 2 and 3 who had yet to receive treatment, a study focusing on secondary outcomes reported. While all participants reported overall satisfaction with posture and physical appearance after surgery, gross ...
Transforming our homes into adaptive living spaces
Sep 5, 2024 · A modern era of wheelchair-accessible time travel is finally here. When we moved into the house in 1995, my rooms were pretty plain. My bedroom had a bed, a chair for my nurses, and my power wheelchair that I drove around during the day. The wheelchair was (and still is) charged in my medical supply room at night.
Hospital stays, costs drop with year's use of Spinraza for SMA: Study
Jul 15, 2024 · 68% decline in total hospitalization costs seen across year of use. Considering all patients collectively, total hospitalization costs decreased by 68% after starting Spinraza, from $1,967,121 to $627,063. The total number of emergency department admissions also declined after patients started on Spinraza, by about 8% on average, and emergency ...