The FDA recently granted EXG-34217 Rare Pediatric Disease Designation (RPDD) and Regenerative Medicine Advanced Therapy (RMAT) Designation.

Newborn testing has long-term heath outcomes, but legislative changes could affect newborn screening effectiveness.

Scientists at the Icahn School of Medicine at Mount Sinai and collaborators have identified novel genetic interactions that may contribute to congenital heart disease (CHD), a common birth defect.

“Educating people about the signs and symptoms of MCT8 deficiency, as well as the journey of patients like my son Colton is critically important,” added Favre. “On Rare Disease Day and every day, MCT8 ...

The University of Texas MD Anderson Cancer Center today was awarded nearly $23 million from the Cancer Prevention and Research Institute of Texas (CPRIT) in support of 20 cancer research projects to ...

OKLAHOMA CITY, OKLAHOMA / ACCESS Newswire / February 20, 2025 / Xironetic announced today the publication of data from a recent study evaluating the potential benefits of augmented reality guidance wi ...