Neuroinflammation triggers autism-like regression in mouse model

Autism spectrum disorder (ASD) is a neurodevelopmental condition estimated to affect approximately 1 in 100 children ...

Filana Therapeutics Announces Publication in Epilepsia of Preclinical Simufilam Data

Filana Therapeutics, Inc. (NASDAQ: FLNA, "Filana Therapeutics", the "Company"), a biotechnology company focused on developing therapies for Tuberous Sclerosis Complex (TSC)-related epilepsy, today ...

Base editing repairs mutation and liver function in mouse model of Zellweger spectrum disorder

In 2025, baby KJ Muldoon became the first person to receive a personalized gene editing treatment, which likely saved his ...

Small molecule drug candidate offers hope for rare kidney stone disease with no current treatment

Scientists at the Buck Institute for Research on Aging have shown that an orally administered small molecule, N-propargylglycine (N-PPG), can completely prevent the formation of calcium oxalate kidney ...

New mouse model of virus-driven liver cancer may boost diagnosis and treatments

Liver cancer is one of the world's deadliest cancers, and most cases are linked to chronic viral hepatitis. Yet scientists have lacked an animal model that faithfully recapitulates how the disease ...
Kaleido Scope

Retinitis pigmentosa mouse models reflect pathobiology of human RP59

One of those genes encodes the enzyme DHDDS, part of the pathway that glycosylates proteins in higher cells. Retinitis pigmentosa from DHDDS mutations is called RP59. This is a recessive genetic ...
Baylor College of Medicine

Researchers at BCM and TCH create mouse model to mimic Parkinson’s disease

Researchers at the Jan and Dan Duncan Neurological Research Institute (NRI) at Texas Children’s Hospital and Baylor College of Medicine recently made an important advance in neurodegenerative disease ...