Jason Mast is a general assignment reporter at STAT focused on the science behind new medicines and the systems and people that decide whether that science ever reaches patients. You can reach Jason ...

Researchers at Oregon Health & Science University have identified a gene that plays a crucial role in the brain's ability to form the protective layer around nerves known as myelin, potentially ...

If administered early, gene therapy has the potential to change the medical history of children born with metachromatic leukodystrophy (MLD), a rare and lethal neurodegenerative disease of genetic ...

Children with MLD treated with arsa-cel were less likely to develop severe motor impairment or die than typical among historical controls. Benefits of the gene therapy persisted over several years of ...

PHILADELPHIA, March 11, 2025 /PRNewswire/ -- In a preclinical study, researchers at Children's Hospital of Philadelphia (CHOP) demonstrated a novel gene therapy with potentially increased ...

SAN DIEGO COUNTY, Calif. — A Carlsbad-based foundation is providing a lifeline for a 3-year-old girl diagnosed with an extremely rare and terminal neurodegenerative disease. The family of Harlow Prado ...

The parents of a little boy diagnosed with a rare brain disease are raising awareness after common cold symptoms led to his diagnosis. Little Arlo Paton was just five months old when he was found ...

A team led by University of Pittsburgh School of Public Health geneticists has shown, for the first time, that a gene "silencer" that resides in junk DNA is directly sparing people from a devastating ...

The conclusions drawn by Zhang et al. may not align with current best practices and could risk creating false hope for MLD families. The Metachromatic Leukodystrophy Initiative (MLDi) critically ...

Leukodystrophy isn’t just one disease; it’s actually a group of diseases that affect the central nervous system. Doctors are discovering new forms of leukodystrophy all the time, but experts currently ...