Latest News

Recall the saying “there are a thousand ways to skin a cat”? Researchers are rephrasing it in regard to the mosquito. Fifty years ago, we thought that the war against malaria was pretty much won. We had quinine and chloroquine, which were effective against the parasite that causes the disease....
ORLANDO — Fox Chase Cancer Center investigators report that a two-drug blockade of mTOR signaling appears safe in metastatic kidney cancer in a phase I trial. Early data suggests that a combination of temsirolimus and bryostatin may be active in patients with rare forms of renal cell cancer, which are...
EL PASO — Two-year-old Emmanuel Gonzalez felt the sun on his face for the first time in weeks Sunday. He has a rare immune system disorder and had to stay in his room at Providence Memorial Hospital while he got well enough to make his first trip down to the...
It’s shortly after 5 a.m. when the phone rings, and on the line is a clearly anxious and worried parent. “Sierra is having a lot of problems tonight,” Shaylene Akery tells a CNN producer. “We have to take her to the hospital, but we still want to talk to you...
GAINESVILLE — A dog born with a deadly disease that prevents the body from using stored sugar has survived 20 months and is still healthy after receiving gene therapy at the University of Florida — putting scientists a step closer to finding a cure for the disorder in children. Called...
The son of Vitaliy Svichynskyi and Maria Svichynska was nearly two months old when he suddenly started to lose the ability to move. The family soon learned their baby boy Dmytro was born with Spinal Muscular Atrophy (SMA) Type 1, the most severe type of the rare genetic disorder found in...
NOVATO, Calif. – Ultragenyx Pharmaceutical Inc., a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare genetic diseases, today announced that the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) application for UX053, an investigational mRNA therapy being...