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.

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.

Far from the Groundhog Day spectacle of amyloid-targeting antibodies that do little to nothing for patients in trial after trial, researchers have reported success in reducing the risk of Alzheimer’s disease (AD) in unexpected ways. Researchers at the Cleveland Clinic Foundation have discovered that a Viagra prescription was associated with a roughly 70% reduced risk of being diagnosed with AD. And investigators from the University of Washington reported that in the prospective Adult Changes in Thought study, a long-term study into risk and protective factors for dementia, persons who underwent cataract surgery reduced their risk of AD by 30%, an effect that lasted for 10 years after surgery.

In the Dec. 3, 2021, issue of Science, researchers reported that a missense variant that is highly enriched in the Amish population was associated with reduced levels of both LDL cholesterol and fibrinogen. To date, very few variants have been identified that affect more than one risk factor for cardiovascular disease.

Far from the Groundhog Day spectacle of amyloid-targeting antibodies that do little to nothing for patients in trial after trial, researchers have reported success in reducing the risk of Alzheimer’s disease (AD) in unexpected ways. Researchers at the Cleveland Clinic Foundation have discovered that a Viagra prescription was associated with a roughly 70% reduced risk of being diagnosed with AD. And investigators from the University of Washington reported that in the prospective Adult Changes in Thought study, a long-term study into risk and protective factors for dementia, persons who underwent cataract surgery reduced their risk of AD by 30%, an effect that lasted for 10 years after surgery.

In the December 3, 2021, issue of Science, researchers reported that a missense variant that is highly enriched in the Amish population was associated with reduced levels of both LDL cholesterol and fibrinogen. To date, very few variants have been identified that affect more than one risk factor for cardiovascular disease.

Investigators at the University of Wisconsin have decreased anxiety-related behaviors in monkeys with an anxious temperament by treating them with a combination of gene therapy and low doses of the atypical antipsychotic drug clozapine, which is approved for the treatment of schizophrenia and schizoaffective disorder.