Novel therapies for translation in childhood diseases

Theme Leads: Professor Francesco Muntoni

Deputy Theme Leads: Dr Karin Straathof and Dr Haiyan Zhou

In this theme we will build our capacity to offer clinical trial options to children affected by rare and complex conditions who have limited treatment options. To do this we will invest in more staff who are trained to deliver specialist treatments as well as recruiting more trial design and regulatory experts. We will be continuing to work closely with patient and public advisory groups to ensure that the patient voice is heard from the very early stages of development of these novel trials.

We also aim to more fully map the characteristics of patients with rare diseases to help us identify new drug targets. By pooling information from novel imaging technologies, and laboratory tests of biological samples, we will be able to monitor disease progression and response to treatments more effectively.

Novel Therapies theme funding call are advertised in the GOSH BRC Novel Therapies Project Adoption Call.