The FDA has awarded rare pediatric disease designation to Orphagen Pharmaceuticals Inc.'s OR-449 for the treatment of pediatric adrenocortical carcinoma (ACC). OR-449 is a selective, first-in-class, potent and orally bioavailable small-molecule antagonist to steroidogenic factor-1 (SF-1; NR5A1), an orphan nuclear receptor and transcription factor that is essential for the growth and development of the adrenal gland.
As expected, the U.S. FDA gave its go-ahead to lecanemab, an amyloid-beta binder for mild cognitive impairment caused by Alzheimer’s disease (AD) and mild AD from Biogen Inc. and Eisai Co. Ltd, which have assigned to the compound the brand name Leqembi.
As expected, the U.S. FDA gave its go-ahead to lecanemab, an amyloid-beta binder for mild cognitive impairment caused by Alzheimer’s disease (AD) and mild AD – together known as early AD – from Biogen Inc. and Eisai Co. Ltd, which have assigned to the compound the brand name Leqembi.
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.
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