SYNGAP1-related disorders (SRDs) are rare neurodevelopmental conditions characterized by a wide range of symptoms, including intellectual disability, epilepsy, motor deficits and increased risk-taking behavior.

A review of 2025's noteworthy advances in medical research, including GLP-1 receptor agonists as anti-aging drugs, tumor-agnostic therapies and xenotransplants.

Multiple sclerosis (MS) is a chronic immune-mediated disease characterized by the destruction of myelin sheaths, neuroaxonal damage, glial cell activation and formation of demyelinated plaques in the CNS. Since MS is considered a prototypic antigen-specific autoimmune disease, restoring immune tolerance to self-antigens is being explored as a therapeutic strategy.

Gene editing technologies are moving forward in preclinical development with innovative strategies designed to treat diseases at their root and even reverse them. However, many approaches still struggle to reach target cells or tissues – either they fail to arrive, or their efficacy is low. In vivo therapies face numerous challenges, but despite these hurdles, 2025 has marked a year of remarkable progress.

Syremis Therapeutics Ltd. has launched with $165 million in series A funding to develop novel medicines for the treatment of mental health disorders.

Researchers at the University of Sydney have uncovered a mechanism that may explain why glioblastoma returns after treatment, and the world-first discovery offers new clues for future therapies. Glioblastoma is one of the deadliest brain cancers, accounting for about half of all brain tumors, with a median survival rate of just 15 months. Despite surgery and chemotherapy, more than 1,250 clinical trials over the past 20 years have struggled to improve survival rates.