By using data collected over decades in a database of more than 10 million active-duty military personnel, researchers have managed to nail down the connection between Epstein-Barr virus (EBV) infection and multiple sclerosis (MS).
Clinicians at the University of Maryland have transplanted a heart from a genetically modified pig bred by Revivicor Inc., a subsidiary of United Therapeutics Corp., into a patient with end-stage heart failure.
Investigators at the University of Freiburg and Swiss startup Ultimate Medicine have identified a compound produced by the gut microbiome as contributing to age-related cognitive decline by modulating inhibitory synaptic transmission and neural network activity.
Researchers at PTC Biotherapeutics Inc. have identified orally available small-molecule compounds that broadly lowered the levels of mutant huntingtin protein in both the brain and the periphery by affecting its splicing. One of those compounds, PTC-518, is currently in phase I trials as a therapy for Huntington's disease.
Attempts to modernize the conceptual framework of brain function and dysfunction are one prerequisite for brain disorders to benefit from precision medicine. For the circuit-based insights that are slowly emerging to benefit patients, though, better targeting methods are needed.
Brain disorders have not yet profited from advances in precision medicine to the same extent that other disorders have. With the advent of magnetic resonance imaging and other technologies, watching the brain at work has made great strides in recent decades. But those data have often been shoehorned into the categories of the Diagnostic and Statistical Manual of Mental Disorders. Researchers are working to bring diagnostic categories in line with a modern understanding the brain.
Two trials presented at the annual meeting of the American Society of Hematology (ASH) showed that treatment with the antithrombin inhibitor fitusiran reduced the bleeding rate in patients with severe hemophilia by roughly 90%, regardless of whether they had type A or B, and whether they had developed antibodies to recombinant clotting factors.
Clonal hematopoiesis of indeterminate potential (CHIP) was associated with a 35% reduced risk of Alzheimer's disease (AD) in studies presented at the plenary session of the American Society of Hematology (ASH) annual meeting on Sunday.
