In this Healio Video Perspective from Clinical Trials at the Summit, Christine Kay, MD, discusses phase 1/2 results for ...

"Everything was orderly and convivial," Hollywood director-writer Jordan Walker-Pearlman told THR after the Bel-Air house ...

Rare diseases affect more than 300 million people worldwide - 36 million in the EU alone - but they are often overlooked due ...

In one of the first major tests of the new FDA leadership’s regulatory philosophy toward gene therapies for rare diseases, ...

The FDA is investigating the deaths of two boys with Duchenne muscular dystrophy who died soon after being treated with ...

Researchers found that at week 24, 81 and 16% of the treated and control wounds, respectively, were at least 50% healed from baseline.

Researchers have developed a gene therapy that significantly slowed motor function loss in preclinical models of amyotrophic ...

Springfield, MO; Passed March 4, 2025 Atascadero, CA Truly Legendary Car Customizer, Hot Rodder and Racer, Father and Friend ...

Pompe's disease is a lysosomal storage disorder caused by a genetic deficiency of GAA. The disease is associated with severe and progressive physical disability and premature death, and patients ...

While some scientists argue that agricultural gene editing regulations are overly restrictive, a new paper from the ...

Researchers say they have finally confirmed the cancer-stopping gene found in elephants and several other animals.

The U.S. Food and Drug Administration said on Tuesday that it is investigating reports of two deaths due to acute liver ...