CAP-002 is the first engineered IV-delivered gene therapy that crosses the blood-brain-barrier and detargets the liver and dorsal root ganglia to enter the clinic THOUSAND OAKS, Calif., May 12, 2025 —...

The greater than three-fold increase in autism diagnoses among students in special education programs in the United States between 2000 and 2010 may be due in large part to the reclassification of ind...

In this perspective, Wątor-Wilk et al. discuss the structural biology of deoxyhypusination, essential for eIF5A/aIF5A activation. Comparing recent crystal and cryo-EM structures across different speci...

Patient advocate Austin Leclaire, who died Feb. 1, played a pivotal role in spurring the development of the first medicines to treat Duchenne muscular dystrophy.

Rod-shaped fragments of RNA called “obelisks” were discovered in gut and mouth bacteria for the first time

America’s best scientific minds are scrambling to determine the impact of the disruption.

Knowledge of the natural history of CDKL5 deficiency disorder (CDD) is limited to the results of cross-sectional analysis of largely pediatric cohorts. Assessment of outcomes in adulthood is critical ...

The Institut de Biologie du Développement de Marseille (IBDM) is inviting applications for group leader positions. We are seeking innovative researchers who aim to address fundamental questions in bio...

NYU Grossman School of Medicine’s Neurogenetics Fellowship focuses on care for complex neurogenetic conditions and groundbreaking research.

The American Epilepsy Society (AES) meeting is the largest epilepsy meeting of the year, and because it takes place every month of December it also serves as an annual review on the understanding and ...