A gene-editing therapy led to durable changes in serum transthyretin levels among people with hereditary transthyretin amyloidosis with polyneuropathy. Polyneuropathy stages and disability scores were ...

A company called uniQure has announced promising results from a trial of a new gene therapy for Huntington’s disease. The news has spread quickly through families affected by this condition, who have ...

Share on Pinterest The new gene therapy showed promise by significantly slowing down Huntington’s disease. Santi Nuñez/Stocksy Huntington’s disease is a genetic brain disorder that causes symptoms ...

An experimental gene therapy was found to slow the progression of Huntington's disease, marking a major step toward a potential first genetic treatment for the condition.

Groundbreaking study demonstrates that targeted oligodendrocyte gene therapy leads to decreased N-acetylaspartate (NAA) levels and increased ...

The first participant in an investigational gene therapy trial of STXBP1 encephalopathy died, Capsida Biotherapeutics said. The death occurred days after dosing, according to information posted on the ...

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 ...

UniQure's therapy, called AMT-130, reduced disease progression by 75% at 36 months in patients who received a high dose.

FOLLY BEACH — His pale blue eyes flashing and sporting a wide grin, Max Harbin toddled forward, leaning on a small cart as he ventured a few steps. His parents, Jim and Laura Harbin, watched him ...

New study data showed treatment slowed signs of disease progression by 75% after three years, a finding that could have “massive effects on patients’ lives,” an investigator said.

This article is part of Nature Outlook: Skin, an editorially independent supplement produced with financial support from LEO Pharma. About this content. KaDee Troop is the mother of seven adopted ...