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网页2024年9月13日 · SMA treatment Zolgensma seen to benefit patients in real world. 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.
Standardized SMA newborn screening may aid early treatment
网页17 小时之前 · Patients commonly started treatment between 2 and 4 weeks of age (66%), with more than half (53%) not starting before the patient was 3 weeks of age, usually due to delays related to health insurance. All but one provider had treated patients with Spinraza (98%), Zolgensma (95%), and Evrysdi (83%). Most (81%) identified Zolgensma as …
SMA treatment Zolgensma seen to benefit patients in real world
网页2024年9月13日 · 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 ...
Surgery for scoliosis slows SMA lung function decline: Study
网页2 天之前 · 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 ...
News – HCP SMA News Today
网页A milder form of spinal muscular atrophy (SMA) was discovered in two siblings who carry a common mutation in one SMN1 gene and a rare mutation in the other, a new study by Mayo Clinic researchers reported. Skin cells from the brother and sister showed higher levels of…. August 23, 2024 News by Patricia Inacio PhD.
Spinraza, in Range of SMA Patients, Shows Efficacy Over Years of Use
网页2020年5月20日 · “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 ...
Spinal Muscular Atrophy: Cardiac Disease – HCP SMA News Today
网页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 ...
HCP SMA News Today – The Web's Leading Resource For People …
网页Treatment with Spinraza (nusinersen) generally stabilized or improved motor function for older patients with spinal muscular atrophy (SMA), according to a review and meta-analysis of previously published observational studies. The new analysis, which spanned a treatment period of up to two years, involved adults and adolescents with….
Top 10 SMA Stories of 2021 – SMA News Today
网页2022年1月3日 · 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 ...
With Zolgensma, Gene Therapy Takes Step Forward in Treating …
网页by Emma Yasinski May 31, 2019. Zolgensma ‘s approval as the first gene therapy for treating all forms of spinal muscular atrophy (SMA) in children up to 2 years old is another step forward — an exceptional and far-reaching one — in the seeming revolution underway in treating more diseases, and an increasing number of rare ones, by ...