New program launched to accelerate access to personalized therapies for children with rare conditions
Today, Wednesday 22 November, the government announced its support for the Rare Therapies Launch Pad, a new programme that will develop a pathway for children with rare conditions to access individualized therapies. The programme’s first project will explore the use of individualized therapies known as antisense oligonucleotides (ASOs) to treat children with ultra-rare and life-threatening … Read more