A gene therapy targeting organs beyond the brain could improve quality of life and possibly extend survival in children with the neurodegenerative disease.
A new porcine model for the rare neurological disorder CLN3 Batten disease more faithfully recreates human symptoms and could accelerate drug development.
Left out of clinical trials for years, preterm infants have very few drugs developed just for them. Regulators, industry, and neonatologists plan to fix that.
Alpha thalassemia was once a fatal diagnosis. Now, a clinical trial tests if administering a mother’s stem cells in utero may cure kids before they’re born.
Malaria continues to drive urgent research worldwide, with new therapies and tools emerging to combat the parasite’s complex lifecycle and global burden.