Two Italian women in their twenties on Tuesday received pioneering gene therapy for rare hereditary blood diseases in Pavia ...

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

This is a developing news story. Please check back soon for updates. The FDA granted VG801 rare pediatric disease designation ...

Glioblastoma has been deadly and hard to treat, but researchers say they’ve made progress with bivalent CAR T-cell therapy ...

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

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

A Falmouth teen, Jayden Wilsey, is the first to receive gene therapy to treat sickle cell at Boston Children's Hospital.

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

Siegfried Priglinger, MD discussed research he presented focused on pediatric patients with inherited retinal diseases with ...

Trump health officials signal eased FDA rules for rare disease gene therapy, but concerns linger over safety, oversight, and ...

Infant With Rare, Incurable Disease Is First to Successfully Receive Personalized Gene Therapy Treatment May 15, 2025 — A research team has developed and safely delivered a personalized gene ...

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