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Cerebral creatine (Cr) deficiency syndromes are caused by AGAT, GAMT or SLC6A8 deficiencies that may cause severe neurodevelopmental delay and intellectual disability.
Communication between adipose tissue and the brain increases the risk of cognitive impairment in patients with insulin resistance through extracellular vesicles (EVs) containing microRNAs (miRNAs). Neurons could be damaged when these nucleotides reach the hippocampus guided by membrane proteins in prediabetic overweight people.
Argininosuccinic aciduria (ASA) is a disorder caused by argininosuccinate lyase (ASL) deficiency and is the second most common inherited urea cycle defect.
The FDA has granted orphan drug designation to Iecure Inc.'s lead product candidate GTP-506, an investigational product for the treatment of ornithine transcarbamylase (OTC) deficiency.
Researchers from the University of Pittsburgh presented preclinical data for novel messenger RNA (mRNA) therapy consisting of human very long chain acyl-CoA dehydrogenase (hVLCAD) mRNA; the product is being developed as a potential treatment of VLCAD deficiency.
The FDA has cleared Immusoft Corp.'s IND for ISP-001 (iduronicrin genleukocel-T) for the treatment of mucopolysaccharidosis type I (MPS I), allowing initiation of a phase I trial.
Five months after winning its first approval in Japan, Sanofi SA’s enzyme replacement therapy, Xenpozyme (olipudase alfa), earned a U.S. FDA nod for use in pediatric and adult patients with acid sphingomyelinase deficiency (ASMD), becoming the first medication designed to treat symptoms not related to the central nervous system.
Glucagon-like peptide 1 (GLP-1) signaling to intraepithelial lymphocyte (IEL) cells in the gut, where most GLP-1 is made, was important for inflammation in the gut itself, but less so for systemic inflammation.
In pursuit of "an opportunity to accelerate the establishment of clinical development and commercial capabilities in the U.K.," Neurocrine Biosciences Inc. said it will buy Diurnal Group plc for about £48.3 million (US$56.5 million). Cardiff, U.K.-based Diurnal is a specialty pharma developing hormone therapies for rare and chronic endocrine conditions. The all-cash transaction represented a 144% premium of the Aug. 26 closing price of Diurnal shares (LSE:DNL). Shares closed 134.7% higher Aug. 30 at £26.40.
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