KJ Muldoon, a 10-month-old baby, was diagnosed with the genetic disease carbamoyl-phosphate synthetase 1 deficiency after he ...

New gene therapy improves the life of 18-year-old with Sickle Cell Disease which affects 5,000 New Jersey residents living ...

In the first part of this series, we explored how early genetic screening and gene therapy transform the lives of newborns ...

As newborn screening and rapid DNA sequencing become routine, we are poised to catch and treat inherited diseases at their ...

Machine learning models have seeped into the fabric of our lives, from curating playlists to explaining hard concepts in a ...

Baby KJ was successfully treated with a customized CRISPR gene editing therapy by a team at Children's Hospital of ...

KJ Muldoon, a 10-month-old baby diagnosed with a rare metabolic disease called CPS1 has been at Children Hospital of ...

Cell and gene therapy (CGT) represents the pinnacle of biomedical innovation, offering unprecedented potential to treat, ...

The company is one of the first to receive a “platform technology designation,” which could speed the review of certain gene ...

After 307 days at the Children's Hospital of Philadelphia, a local baby who was born with a rare and usually fatal disorder ...

FDA alignment on outcomes for the key Part B trial was supported by caregiver-reported milestone gains. Taysha Gene Therapies ...

KFSHRC accounts for 52.4% of all active clinical trials in Saudi Arabia.