A two-and-a-half-year-old girl shows no signs of a rare genetic disorder, after becoming the first person to be treated for ...

A groundbreaking gene therapy has restored sight in four young children born with severe blindness due to a rare genetic ...

Pfizer has ended the global commercialisation of its haemophilia B (factor IX deficiency) gene therapy Beqvez, citing soft ...

In a major breakthrough, four children born with severe visual impairments have experienced life-changing improvements in their ...

As Rare Disease Day is commemorated on 28 February, experts discuss the barriers to gene therapy access for some rare ...

The global Gene Therapy market, valued at US$7.21 billion in 2023, is forecasted to grow at a robust CAGR of 19.4%, reaching ...

Four young children have gained life-changing improvements in sight following treatment with a pioneering new genetic ...

The children were born with a severe impairment to their sight due to a rare genetic deficiency that affects the AIPL1 gene. The condition, a form of retinal dystrophy, means those affected are born ...

YolTech's gene therapy is intended as a one-time life-long cure for patients living with PH1, which can lead to kidney ...

The children were born with a severe impairment to their sight due to a rare genetic deficiency that affects the AIPL1 gene. The condition, a form of retinal dystrophy, means those affected are ...