News

Riley's miracle: three open-heart surgeries by age nine
Meet Riley, a remarkable boy who has undergone six heart surgeries by the age of nine. His inspiring story of resilience and ...
Lab-grown 'tiny hearts' bring hope for children and adults with genetic heart disease
Scientists from QIMR Berghofer's Cardiac Bioengineering Lab have developed lab-grown, three-dimensional heart tissues known ...
Gene therapy restores hearing in toddlers and teenagers born with congenital deafness – new research
This is the first time such results have been achieved in both children and adult patients born with a specific type of ...
Mouse models for ultra-rare disorder could pave the way for nervous system gene editing therapies
Scientists at The Jackson Laboratory (JAX) have developed mouse models that survive premature death and enable pre-clinical ...
Penn and CHOP open a center for a rare genetic condition, thanks to a $25 million donation
Michael Armellino first heard of Williams syndrome when his partner’s granddaughter, Maelyn, was born with the rare genetic condition 10 years ago. A simple internet search laid out that she could be ...