KJ Muldoon's life-threatening disorder, called severe carbamoyl phosphate synthetase 1 (CPS1) deficiency, was treated with ...

A 5-year-old boy from Center City has faced rare odds since birth and now this warrior is in a real life race to rewrite his story.

A new review sheds light on the complex molecular mechanisms behind Angelman syndrome (AS), a rare neurogenetic disorder, and ...

Repurposing existing therapies may help slow muscle weakness in patients with Duchenne muscular dystrophy, a new study ...

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

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

In this video, Jay Chhablani, MD, discusses preliminary data from the ArMaDa study of OCU410 gene therapy for geographic atrophy presented at the ARVO meeting.

Scientists at the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), Milan, have found that gene editing using ...

Needham raised its price target for Taysha Gene Therapies Inc. (NASDAQ:TSHA) to $8 from $6, while maintaining a Buy rating on ...

Scientists at the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) , Milan, have found that gene editing using CRISPR-Cas9 in combination ...

Discover how biotechs and big pharma are developing novel treatments for high cholesterol with a range of different targets.

Pepgen will stop development of its Duchenne MD exon-skipping therapy PGN-ED051 after increases in dystrophin levels proved ...