pediatrics

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

Enzyme replacement therapy has been the mainstay of Pompe disease. New and emerging therapies may provide more options for patients.

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

In some kids, an RSV infection is mild, while others are hospitalized. Natural killer cells might be the reason why.

Epilepsy onset and seizure severity are linked to variants in the SCN2A gene, potentially leading to improved diagnostics and treatments.

This year’s laureates have made groundbreaking discoveries from unlocking the secrets of DNA to fighting enteric diseases and cancer.

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.

Scientists developed a gel formulation that makes swallowing medicines easier.