Neuroscience

A statistical model using two measures of brain activity could anticipate if people given an injection would be in severe pain for the next month.

In a Phase 2b trial, Khondrion’s sonlicromanol led to significant health benefits for patients with mitochondrial disease linked to a common mutation. 

A gene therapy targeting organs beyond the brain could improve quality of life and possibly extend survival in children with the neurodegenerative disease.

Unlike in other brain regions, levels of the neurotransmitter decrease in the cerebellum when anxiety is high, suggesting a potential new drug target.

Vertex Pharmaceuticals’ drug, Journavx, blocks a sodium channel outside of the brain. Experts hope that this non-addictive pain drug may be the first of many.

People and mice with ALS-linked mutations show altered sleep patterns early in life. Modifying sleep hormone levels normalized sleep cycles in mice.

Levels of neuroactive steroids during the third trimester of pregnancy may indicate postpartum depression risk.

Targeting a gut bacterial species or its metabolites could potentially improve diabetes management by reducing sugar preferences. 

Lundbeck Therapeutics is testing a monoclonal antibody that targets a novel neuropeptide in a Phase 2b clinical trial. 

The protein APLP1 strongly associates with brain-derived EVs. A blood test could one day isolate these molecular packets to diagnose neurological disorders.

The gene therapy treats the rare genetic disease AADC deficiency by delivering a functional gene into the brain.

Researchers developed a new pill with promising applications for treating pain due to gastrointestinal disorders.

Industry leaders predict the most exciting advances in drug discovery and development in the coming year.

Genetic analysis of organoids and brain tumors showed that the cancers are packed with cells that haven’t reached a mature state.

An oligonucleotide transport vehicle could transform neurological disease treatment by enhancing drug delivery across the blood-brain barrier.

The FDA approved two new medications for Niemann-Pick disease type C that improve symptoms, showing the power of community advocacy.

New research revealed that manufacturer-funded clinical trials deem drugs more effective than non-sponsored trials.