SMA News Today Home | SMA News Today
Nov 6, 2024 · Changes in various genes tied to key processes affect SMA’s course: Study. Variations in more than a dozen genes related to inflammation, neurodevelopment, and oxidative stress associate with the severity of spinal muscular atrophy (SMA) and disease type, as well as person’s age at onset and their motor and lung function, researchers report.
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.
Zolgensma most successful in presymptomatic SMA infants: Study
4 days ago · Zolgensma delivers working copies of SMN1 to body’s cells. Zolgensma (onasemnogene abeparvovec-xioi) is a one-time gene therapy that delivers working copies of SMN1 to the body’s cells so that functional SMN protein can be produced.. In the current study, researchers from Germany, Austria, and Switzerland examined real-world data from 343 SMA patients treated with Zolgensma, including ...
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 ...
Top 10 SMA Stories of 2022 - SMA News Today
Dec 29, 2022 · No. 6 — “#MDA2022 – ‘Amazing’ Walking Ability Seen in SMA Children on Spinraza” The Phase 2 NURTURE trial (NCT02386553) is testing the injectable therapy Spinraza in 25 children genetically diagnosed with SMA, who started treatment before the age of 6 weeks, prior to showing any symptoms. At the Muscular Dystrophy Association annual meeting, researchers presented the trial’s ...
Top 10 SMA Stories of 2021 - SMA News Today
Jan 3, 2022 · No. 1 — “ A ‘Miracle’: Spinraza Gives Boy, Now 5, a Chance to Grow Up ”. Our most-read article of 2021 concerned the story of 5-year-old Jase Yoder, who initiated Spinraza treatment in the Phase 2 NURTURE trial (NCT02386553) just 18 days after being diagnosed with SMA type 1. Starting treatment when he was not yet 1 month old, Jase ...
HCP SMA News Today – The Web's Leading Resource For People …
Oct 10, 2018 · SBMA therapy NIDO-361 granted orphan drug status in Europe. NIDO-361, Nido Biosciences’ experimental oral treatment for spinal and bulbar muscular atrophy (SBMA) — a disease type marked by progressive weakness in the muscles of the throat and mouth, and those closer to the trunk —…. October 21, 2024 News by Lindsey Shapiro PhD.
New large-scale SMA carrier screening test most effective: Study
Oct 14, 2024 · A new large-scale genetic screening technique for spinal muscular atrophy (SMA) called comprehensive SMA analysis, or CASMA, was more effective at detecting disease-related mutations in carriers than standard approaches, a study reports. CASMA “is a simple and accurate screening method for SMA, which shows greater clinical efficacy in the ...
Phase 3 trial of apitegromab in SMA type 2, 3 patients now...
Sep 20, 2023 · The Phase 3 SAPPHIRE trial (NCT05156320) aimed to recruit up to 204 patients with SMA type 2 or 3, who are on stable treatment with Spinraza (nusinersen) or Evrysdi (risdiplam) at over 50 sites across the U.S. and Europe.. Its main goal is to evaluate the effectiveness of one year of apitegromab treatment versus a placebo, using a standardized measure of motor function called the Hammersmith ...
Spinraza, in Range of SMA Patients, Shows Efficacy Over Years of Use
May 20, 2020 · “As the first approved treatment for SMA, Spinraza offers a significant data set that allows us to uniquely assess the safety and durability of repeated doses over time … across age groups and varying disease severity,” Alfred Sandrock, Jr., MD, PhD, executive vice president, Research and Development at Biogen, said in a press release.. SHINE (NCT02594124) is an ongoing Phase 3 open ...