Meet the researchers shaping the future of children's healthcare

Every day at Great Ormond Street Hospital (GOSH), children and families arrive looking for answers. Some are living with rare conditions that are difficult to diagnose. Others are waiting for treatments that do not yet exist.

Behind every breakthrough in children's healthcare is a researcher asking a question, testing an idea, or developing a new approach that could change a child's future.

But the scientists and clinicians who will lead tomorrow's discoveries for children with rare disease often face some of the biggest challenges securing the funding they need to develop their ideas.

That's why GOSH Charity is investing in five researchers over the next three years through the Emerging Leaders Fellowship. Working across paediatric rare diseases at UCL Great Ormond Street Institute of Child Health and GOSH, these researchers are tackling some of the most pressing questions facing patients and families today.

From finding clues that build disease understanding to developing treatments, meet the five Emerging Leaders Fellows helping to shape the future of children's health.

"You often feel like a detective, trying to understand the underlying cause of complex conditions by piecing together information from the child and their clinical history.”

Amy searches for the genetic clues behind severe epilepsy in young children. By helping families get answers sooner and understanding how these genetic changes affect the brain, her research could pave the way for more personalised treatments that target the cause of epilepsy, not just the seizures.

“I’ve always been fascinated by the brain – how it develops, how it functions and all the complex things it does. Curiosity was really what drove me into this field.”

Naresh is exploring how the brain develops to understand when and how neurological conditions begin. By identifying the earliest signs of something not developing as it should, his research could help children receive a diagnosis and treatment sooner, when it can make the biggest difference.

“Support like this fellowship funding is hugely important. It gives researchers the stability to progress our work and grow our team.”

Jennie is developing new gene therapies for children with genetic kidney diseases. Her research aims to slow or stop these conditions before they lead to kidney failure, helping more children avoid or delay dialysis and kidney transplants so they can spend more time living life beyond the hospital.

Her team recently published new research for a pioneering treatment that can slow a rare genetic kidney disease before kidney failure develops.

“The discoveries we make in rare diseases like EB have the potential to benefit many more patients with more common conditions.”

Rob is turning discoveries in the lab into potential new treatments for children with epidermolysis bullosa (EB), a rare condition that causes the skin and other tissues, such as the airways, in the body to blister and scar easily. His research aims to replace a faulty protein that helps keep these tissues strong, protecting children's airways and reducing inflammation and scarring.

“One day I’ll be seeing patients in clinic, and the next I’m in the lab looking at their genetic data – there’s a really beautiful synchrony between the two parts of my job.”

Maanasa brings together what she learns in the clinic and the laboratory to improve care for children with rare genetic skin conditions that can cause parts of the body to become enlarged or overgrown. By understanding the gene changes behind these conditions and how they develop over time, she's working towards more accurate diagnoses, better information for families and new targeted treatments.

As part of our £70 million Research Strategy, GOSH Charity is investing in the people who will shape the future of child health research.

The Emerging Leaders Fellowship aims to tackle a key bottleneck in the research pipeline: the critical stage where researchers need protected time, stability and support to establish independent research programmes. This fellowship helps to ensure that emerging talent continue to develop, pursue bold ideas and accelerate discoveries that could transform the lives of seriously ill children.

At GOSH Charity, we believe that every child deserves the best chance, starting with research. Research brings hope for answers, better treatments and a healthier future for children, young people and their families.

We look forward to supporting these five fellows’ journeys.