Tilly Dowler, from Castle Donington, Derbyshire, was diagnosed with Stargardt disease, a rare genetic eye condition that causes loss of central vision when she was 15 years old. Symptoms usually ...

so approval in Stargardt disease would prompt the pricing negotiation mechanism. Alnylam's earlier plan was to start trials of the drug before the end of this year in the genetic eye disease ...

At Prime Minister’s Questions, Sir Keir joked that the golden retriever did not look ‘particularly interested’ in what he had ...

The antimalarial drug mefloquine could help treat genetic diseases such as cystic fibrosis, Duchenne muscular dystrophy, as well as some cancers. In these diseases, a mutation in the genetic code ...

Ray Therapeutics, a biotechnology company developing optogenetic gene therapies for vision restoration, today announced it has been awarded an $8 million grant from the California Institute for ...

Parliament’s pup is often spotted sleeping in the Commons near to the MP for Torbay, who has a genetic eye condition called Stargardt disease. Prime Minister Sir Keir Starmer speaking during ...

To mark upcoming Rare Disease Day, pharmaphorum asked a panel ... Foundation Fighting Blindness did with the ProgStar study in Stargardt disease. More importantly, foundations help us understand ...