The department is heavily involved with clinical research, focusing on developing new therapies for children with rare metabolic disorders. Research studies have included observational studies as well as therapeutic clinical trials, ranging from early phase studies to Phase III clinical trials. The Willink Metabolic Unit currently has several clinical trials running for lysosomal storage disorders in conjunction with the Children’s Clinical Research Facility in the Royal Manchester Children’s Hospital.
The department has a strong track record in commercial clinical trials and has delivered several such studies conducted at the NIHR Wellcome Trust Children’s Clinical Research Facility. Many of these have been international, multi-centre clinical trials of enzyme replacement therapies for lysosomal storage disorders, and research undertaken in the department has contributed to the licensing of two such therapies within the last three years.
The research team have also conducted investigator-led clinical trials sponsored by Manchester University NHS Foundation Trust.