When cancer cells in male patients and immune cells in their tumors both lose the Y chromosome, those patients tend to ...

You may have seen it in the news recently: a baby in Pennsylvania with a rare genetic disorder was healed with a personalized treatment that repaired his specific genetic mutation.

Severe autism is considered level 3 on the spectrum, as it profoundly impacts an individual’s behavior and social life. Learn ...

Beam Therapeutics BEAM announced that the FDA has granted an orphan drug designation to its investigational genome-editing ...

Retail buzz around Taysha Gene Therapies Inc surged Tuesday after the company released new clinical results from its TSHA-102 ...

PKD is a genetic disorder marked by kidney cysts and progressive damage. Discover the science, treatment guidelines, and ...

KJ Muldoon's life-threatening disorder, called severe carbamoyl phosphate synthetase 1 (CPS1) deficiency, was treated with ...

A 5-year-old boy from Center City has faced rare odds since birth and now this warrior is in a real life race to rewrite his story.