A five-year-old boy who received the world's most expensive drug as a baby has made "incredible progress" and can walk ...

The first infant to undergo gene-editing therapy has accomplished another first — his first steps. KJ Muldoon is walking and ...

A miracle baby who was treated with a pioneering gene-therapy at CHOP has reached a major milestone as he took his first steps.

A baby born with a rare genetic disorder has taken his first steps after receiving a groundbreaking, personalized gene editing treatment at CHOP.

Medcare Women & Children Hospital has successfully administered a pioneering intrathecal gene therapy for Spinal Muscular ...

This story is part of a series on the current progression in Regenerative Medicine. In 1999, I defined regenerative medicine as the collection of interventions that restore tissues and organs damaged ...

AMD Phase 3 timing, Otsuka funding to 2028, and mixed 4D-710 data with financial risk. See why FDMT stock is a hold.

Revolutions in gene therapy are rapidly changing the landscape of modern medicine. Revolutions in gene therapy are rapidly changing the landscape of modern medicine, forcing society and science alike ...

X-linked retinoschisis gene replacement therapy, aiming to improve retinal architecture and visual function in young male ...

A long-term, trust-based CDMO partnership can support cell and gene therapy developers from early process development through GMP manufacturing and commercial supply.

Kavita Mehta suffers from beta thalassaemia, a genetic condition preventing her body from producing sufficient haemoglobin ...

While gene therapy for cystic fibrosis is still in the research phase, researchers aim to correct the defective gene responsible for the disease. Current research is promising. Cystic fibrosis (CF) is ...