Insulinoma sequencing shows chromosomal aberrations but no driver mutations, suggesting the long-term effects of congenital glucose-galactose malabsorption.

If future studies confirm the rescued receptors work normally, the study offers a new roadmap for rare disease drug development.

CAMBRIDGE, Mass. and SAN FRANCISCO, Sept. 17, 2025 /PRNewswire/ -- The laboratories of Dr. Kevin Bender and Dr. Nadav Ahituv ...

Scientists from the Gray Faculty of Medical & Health Sciences at Tel Aviv University introduced an innovative gene therapy ...

A new clinical trial has revealed encouraging results for a muscle-targeting therapy aimed at improving motor function in ...

When brain development gets off to a bad start, the consequences are lifelong. One example is a condition called SCN2A ...

Scientists have identified a potential target for a new class of drugs that could effectively treat psychiatric disorders, ...

In the search for answers about Alzheimer’s disease, researchers are taking a close look at the immune system of the brain. A ...

In recent years, the U.S. Food and Drug Administration (FDA) has approved a wave of new therapies, including gene therapies, ...

A genetic deletion responsible for the rare “salmiak” pattern in cats has been identified, revealing new information about ...

FDA grants Priority Review to idebenone for Leber Hereditary Optic Neuropathy (LHON), a disease for which there are no existing approved ...

One fundamental feature of neurodegenerative diseases is a breakdown in communication. Even before brain cells die, the ...