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Nov 20, 2024 · Liver-targeting gene therapy lowers mice whole-body SMA symptoms. Gene therapy that selectively delivers the SMN1 gene to the liver reduced alterations related to spinal muscular atrophy (SMA) in a mouse model, restoring …
Taldefgrobep alfa helps counter SMA muscle atrophy in …
6 days ago · Biohaven ran a global Phase 3 clinical trial called RESILIENT (NCT05337553) to test taldefgrobep alfa against a placebo in more than 200 people with SMA, ages 4 to 21, who were on disease-modifying treatment. The study’s main goal was to see if taldefgrobep alfa would improve motor function as assessed by scores on the Motor Function Measure (MFM-32) scale after about a year of treatment.
Liver-targeting gene therapy lowers mice whole-body SMA …
Nov 25, 2024 · Effects of gene therapy that targets the liver in SMA. SMA mice that received the liver-specific gene therapy had increased weight, neuromuscular coordination, and lived longer than the untreated SMA mice, who displayed significant fat buildup in their liver and had elevated triglyceride levels, a sign of poor liver functioning and fatty liver disease.
SMA infants show loss of spinal motor, sensory neurons: Study
4 days ago · Thoracic neurons. SMA is caused by mutations in the SMN1 gene that result in low or no production of the survival motor neuron protein, or SMN. The loss of this protein particularly affects motor neurons, the nerve cells that control voluntary movements. These are progressively lost, leading to muscle weakness and wasting and other disease symptoms.. The disease affects lower motor neurons ...
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.
HCP SMA News Today – The Web's Leading Resource For People …
Hope on the Hill event celebrates progress in SMA research, advocacy. At Cure SMA’s 13th annual Hope on the Hill event in Washington, D.C., spinal muscular atrophy (SMA) patients, their families, lawmakers, and other advocates gathered to celebrate recent progress in disease research and legislative…
Zolgensma, 1st Gene Therapy to Treat SMA, Approved for …
May 24, 2019 · The FDA decision was based on data from the ongoing Phase 3 STR1VE trial (NCT03306277), and the completed Phase 1 START trial (NCT02122952) evaluating the efficacy and safety of Zolgensma, given as an intravenous infusion, to type 1 infants up to 6 months old.By approving Zolgensma to treat pre-symptomatic newborns through toddlers, the FDA went a bit beyond the approval request filed in ...
Accurate genetic testing 'crucial' for early diagnosis of SMA: Study
Nov 15, 2024 · The gold standard for an SMA diagnosis is genetic testing to identify disease-causing mutations. However, “ routine genetic testing methods may overlook structural variants outside of exon 7, potentially leading to misdiagnosis of SMA patients,” the researchers wrote. In this study, the clinicians detailed the cases of two children in the Tianjin area of northern China who had delayed SMA ...
Low bone density prevalent in older SMA children: Study in China
Oct 25, 2024 · Overall, 28.8% of the patients, or 19 of 66 children, were considered to have low BMD, while the remaining 47 children had normal BMD. By SMA type, 7.1% of children with SMA type 1 had low BMD (1 of 14 children), as well as 35.1% of those with SMA type 2 (13 of 37 children) and 33.3% with SMA type 3 (5 of 15 children).. Age was found to be the only clinical factor that significantly linked to ...
SMA treatment Zolgensma seen to benefit patients in real world
Sep 13, 2024 · The team analyzed data from 19 children — more than half of them boys — meeting these criteria, who were participating in RESTORE (NCT04174157), a real-world, observational SMA patient registry tracking treatment patterns and outcomes for up to 15 years.. One patient lived in Israel, while the remaining 18 lived in the U.S.