KJ Muldoon, an infant with a life-threatening metabolic disorder who received a pioneering gene-editing therapy at Children’s Hospital of Philadelphia, went home Tuesday after 307 days at the hospital ...

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 ...

Beam Therapeutics BEAM announced that the FDA has granted an orphan drug designation to its investigational genome-editing ...

After 307 days at CHOP, KJ was dressed in what early studies suggest are the world’s smallest and cutest graduation ...

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

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

Alzheimer’s disease (AD) is a progressive neurodegenerative disorder characterized by synaptic dysfunction, neuronal loss and the accumulation of amyloid plaques and neurofibrillary tangles, ...

Discover the major highlights from ASCO 2025, according to researchers and drug developers who attended the convention.

A baby born with a rare and dangerous genetic disease is thriving after getting an experimental gene editing treatment made ...

Scientists behind a world-first gene therapy reveal how they teamed up with experts across academia and industry to produce a ...

Dr. Angela Jia discussed How Better Treatments and Better Selection Can Improve Patient Outcomes in Localized Prostate Cancer. Dr. Jia discussed two important abstracts presented during the session: ...