Rhabdomyosarcoma (RMS) occurs in 3% of all pediatric cancers. Aberrant Hedgehog (Hh) activation can be ligand-dependent or -independent, but current clinical Hh inhibitors targeting SMO are effective only for ligand-independent tumors. Researchers at Vall d’Hebron Institut de Recerca, Barcelona, Spain, developed a targeted therapeutic for Hh ligand-dependent tumors.
Researchers from Astellas Pharma Inc. presented preclinical data for the novel 5-HT5A receptor antagonist, ASP-5736, being developed for the treatment of fragile X syndrome (FXS).
Metriopharm AG has received a grant of €125,000 from charity Duchenne UK that will support development of the company's lead compound MP-1032 (luminol sodium salt) for the treatment of Duchenne muscular dystrophy (DMD).
A germline change in a single nucleotide increased the risk by up to 6-fold of developing an isocitrate dehydrogenase (IDH) mutant low-grade glioma. The rs55705857 genotype could serve as a biomarker before surgery to identify an early glioma.
Mitochondrial disorders have shown phenotypic and genetic heterogeneity; cytochrome c oxidase assembly protein COX11 is an assembly factor that works as a copper chaperone. Previous findings have unveiled that COX11 knockdown causes a reduction in ATP production in vitro.
Insulet Corp. got the U.S. FDA’s nod to extend use of its Omnipod 5 automated insulin pump to children aged 2 years and up with type 1 diabetes. The expanded indication, just seven months after FDA cleared Omnipod 5 for patients 6 years and older, represents the first time a tubeless automated insulin delivery (AID) system has been authorized for use in children this young.
Health care-focused private equity firm Archimed Group agreed to acquire Natus Medical Inc. for $33.50 per share, a nearly 29% premium over the April 14 closing share price for the provider of medical devices to diagnose and treat neurological and sensory disorders. The deal will take Natus (NASDAQ:NTUS) private after more than 20 years as a publicly traded company.
Engineers at Atlanta-based Georgia Tech’s Center for 3D Medical Fabrication have developed a 3D-printed tracheal splint as an airway for children born with a rare condition known as tracheal agenesis or the absence of a trachea or windpipe.
Jelikalite Corp. has received a breakthrough device designation from the FDA for its wearable neurostimulation device Cognilum. The home-based system aims to reduce symptoms of moderate to severe autism spectrum disorder in pediatric patients aged 2 to 6 years. The designation was based on the results of a double-blind, placebo controlled clinical trial of 30 pediatric patients with moderate to severe autism, which showed the technology can significantly improve symptoms.
Memed Diagnostics Ltd. has published new data from an EU Commission funded clinical trial evaluating its blood test for differentiating bacterial and viral infections. The prospective, multicenter cohort study, titled “AutoPilot-Dx,” was carried out in emergency wards in Italy and Germany. The goals of the study were to validate the performance of Memed’s BV test in a broad pediatric population with respiratory tract infections or fever without source and estimate its potential to impact antibiotic use.
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