Tag: Giant Axonal Neuropathy

Gene therapy offers hope for giant axonal neuropathy patients

sarah JonasResearch Results

A gene therapy developed by researchers at UT Southwestern Medical Center for a rare disease called giant axonal neuropathy (GAN) was well tolerated in pediatric patients and showed clear benefits, a new study reports. Findings from the phase one clinical trial, published in the New England Journal of Medicine, could offer hope for patients with this rare condition and a host of other neurological diseases.

Researchers uncover a potential genetic marker associated with better survival outcomes in patients with head and neck cancer

sarah JonasResearch Results

Researchers from the UCLA Health Jonsson Comprehensive Cancer Center show for the first time that a gene usually linked to giant axonal neuropathy, a rare and severe neurological condition, also plays a role in inhibiting aggressive tumor cell growth in head and neck cancers.