This year saw notable progress in head and neck cancers, Huntington's disease, personalized genetic therapy, and heart ...

U.S. Health Secretary Robert F. Kennedy Jr. on Tuesday added two rare genetic disorders, Duchenne Muscular Dystrophy and ...

The nonprofit is funding research at two universities—Oregon Health & Science University and UMass Chan Medical School—on a ...

Scientists at the Icahn School of Medicine at Mount Sinai have developed a novel artificial intelligence tool that not only ...

Wilson’s disease is an autosomal recessive disorder caused by ATP7B mutations, leading to copper accumulation in the liver, ...

The treatment uses a modified, harmless virus to deliver healthy genes to a patient’s cells to treat genetic diseases. Read ...

A rare genetic disorder discovered by UT Southwestern Medical Center researchers and their colleagues can cause brain damage ...

The FDA has issued its stamp of approval to a new, cell-based option to treat Wiskott-Aldrich syndrome (WAS), marking the ...

This year, gene-editing technology was customized to fix mutations in a single patient’s genes for the first time.

The donation from Don and Anne Edwards aims to speed research, treatment and diagnosis for children with rare and genetic ...

This holiday season, a Lakeland family is wishing to go to North Carolina. It is not for a vacation, but instead an intensive ...

PEWAUKEE, Wis. - A Pewaukee restaurant held a fundraiser on Saturday for a family whose 13-year-old son has an ...