The family of a young girl who has an extremely rare disease has reached their goal of raising money for her initial ...

We’re in the midst of a cancer crisis. More young people being diagnosed. And while the treatments can be life-saving, years ...

A new study links mitochondrial dysfunction to Crohn’s disease, shifting how scientists approach treatment and opening doors ...

In December 2023, through the development efforts of CRISPR Therapeutics and Vertex Pharmaceuticals, their decades-long ...

More than 30 million people in the United States are affected by rare diseases, many of which are life-threatening conditions ...

Johnson & Johnson, a leader in immunology, has developed ARGES, a new AI-powered tool that could transform endoscopic ...

A new discovery offers hope for Huntington’s disease. This discovery provides hope that a DNA repair process may help slow or ...

The FDA has approved Ctexli for the treatment of cerebrotendinous xanthomatosis (CTX), a rare lipid storage disease, in ...

Neglected Tropical Diseases (NTDs) remain a significant health burden in tropical and subtropical regions, with limited ...

Doctors harvest stem cells from the patient’s bone marrow and then send them off to a lab, where CRISPR is used to turn up the production of another, functional version of haemoglobin. This version is ...

CBS 8's Elizabeth Sanchez reports on the family that raised enough money for their 3-year-old girl to begin treatment for a ...