Researchers at the University of Rochester have described a neuroimaging-based biomarker that could identify individuals with early psychosis, and improved their identification when it was added to a standard neurocognitive diagnostic test. In a group of roughly 160 participants in the Human Connectome Early Psychosis Project, individuals who were in the early stages of psychosis had stronger connections from the thalamus (a midbrain sensory processing area) to the cortex, but weaker connections between different cortical areas, than controls.

Our immune cells are not just “defenders” against deadly viruses and pathogens but also a great balancer for tissue homeostasis. For neurological disorders, understanding the neuro-immune axis could be key to treating previously untreatable conditions such as autism spectrum disorder, according to Jun R. Huh, professor of immunology at Harvard Medical School.

The collaboration aims to identify a small molecule that targets mutant androgen receptor (AR) mRNA splicing and causes selective destruction of the disease-causing mRNA.

Sarepta Therapeutics Inc. has signed a licensing and collaboration agreement with Arrowhead Pharmaceuticals Inc. to obtain exclusive global rights to multiple clinical, preclinical and discovery-stage programs for rare genetic diseases of the muscle, central nervous system (CNS) and the lungs. The deal includes ARO-DUX4, ARO-DM1, ARO-MMP7 and ARO-ATXN2.

Enveric Biosciences Inc. has completed preclinical pharmacokinetic (PK) studies of EB-003 in rats and dogs, further supporting EB-003’s oral bioavailability and targeted nonhallucinogenic profile. EB-003 is a neuroplastogen designed to promote neuroplasticity without inducing hallucinations in patients with difficult-to-address mental health disorders.

Oxford Nanopore Technologies Ltd. has established a new collaboration with UK Biobank to create the world’s first comprehensive, large-scale epigenetic dataset. The project will utilize Oxford Nanopore’s DNA/RNA sequencing technology to map the epigenome of 50,000 blood samples from UK Biobank to unlock insights into disease mechanisms, with the aim of improving patient outcomes.

At the Breakthroughs in Muscular Dystrophy special meeting held in Chicago Nov. 19-20, 2024, and organized by the American Society of Gene & Cell Therapy (ASGCT), multiple interventions at the RNA level were among the approaches that were presented to fight muscular dystrophies.