Rare disorders

Contemporary childhood

Our clinical work
Our Centre of Interventional Paediatric Psychopharmacology and Rare Diseases (CIPPRD) provides whole-person medicine and integrated mental and physical health care using clinically cost-effective and innovative interventions alongside patient involvement for children and young people with complex, severe, and treatment-resistant co-occurring neuropsychiatric disorders and medical conditions including rare diseases.

Our service provides highly specialist assessment, pharmacological and psychological treatment for children with a variety of complex neuropsychiatric disorders. This is in the context of neuroinflammatory, metabolic, genetic or neurodegenerative disorders and acquired brain injury; psychotropic-induced side-effects and neuropsychiatric aspects in children with terminal illness. We have specific programs for those with emotional behavioural autonomic dysregulation (EBAD), Rett syndrome, Mucopolysaccharidoses, and other rare disorders.

Our research
Our specialist Centre for Interventional Paediatric Psychopharmacology Research Team (CIPPRT) focuses focusses on translational research, meaning bringing research in the lab to the bedside. This includes clinical trials in rare diseases; developing Patient Centred Outcome Measures (PCOMs) for clinical situations, reviewing medications and profiling symptoms.

Our research also focuses on the effects of drugs on the mind and behaviour and on neurodegeneration in a number of rare disorders for children and young people. For children and young people with co-occurring developmental disorders we also study the best means of assessment and management of complex multiple conditions.

Our research
We collaborate extensively and develop research questions from patients seen in clinics. We also benefit from quick transfer of information from lab to clinical practice due to our joint and cross-working between the teams.

Our significant achievements include having led and completed the first clinical trial in Rett syndrome within the UK in the world’s first Centre for Personalised Medicine for Rett Syndrome (CPMRS), ensuring NHS patients can access new treatments, and then successfully receiving commercial research grants for further Rett clinical trials.

Find out more
Read more about our research and key publications here.

Our future vision for children and young people
We will continue to conduct pioneering clinical trials in rare disorders such as Rett syndrome, to develop the groundwork for implementing transformative clinical trials such as gene therapy when it becomes available.

Meet the team

Dr. Paramala Santosh

Dr. Kitty Kwan

Dr. Ruksana Ahmed

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