Novel therapies for translation in childhood diseases

Theme Lead: Professor Francesco Muntoni

Deputy Theme Lead: Professor Paul Gissen

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: GOSH Biomedical Research Centre Open Theme Meeting

11am – 1pm, Monday 27 November, Lower Ground Seminar Room, ICH. 

GOSH staff are invited to join Professor Franceso Muntoni and Professor Paul Gissen to find out about the recent and upcoming developments in Novel Therapies research at GOSH, funding calls and other opportunities to get involved in your Biomedical Research Centre (BRC). This research theme builds our capacity to offer clinical trial options for children who have limited treatment options and to more fully map the characteristics of these patients to help us identify new drug targets. The meeting is open to anyone with a clinical or research interest in this area.

The Novel Therapies theme is currently advertising a project adoption funding call. Make your application now.

Latest research

Learn about the latest research from the Novel therapies for translation in childhood diseases theme

Funding boost to develop novel therapy for rare metabolic condition

GOSH and UCL Great Ormond Street Institute of Child Health (ICH) researchers Professor Paul Gissen and Dr Julien Baruteau will be part of a new collaboration with Evox Therapeutics that aims to develop a new therapy for the life threatening metabolic condition, argininosuccinic aciduria (ASA)

Precision medicine improves growth for children with a rare form of rickets

A new, targeted drug has been shown to significantly improve growth and control of rickets in children with X-linked hypophosphataemic rickets (XLH), a serious condition affecting about 300 children in the UK.

XLH is caused by genetic mutation that leads to excess activity of fibroblast...