Wellcome HRB Clinical Research Facility

At Wellcome HRB Clinical Research Facility in Dublin, research in congenital and hereditary diseases focuses on Fragile X syndrome, with studies aimed at easing behavioural symptoms and improving everyday functioning for children, adolescents, and young adults.

• Testing a new therapy for Fragile X syndrome and related behavioural challenges
• Evaluating treatment effectiveness in a neurodevelopmental disorder
• Supporting better symptom control for patients and families

This work reflects a broader interest in genetic and developmental conditions, where clinical trials are exploring whether new approaches can improve quality of life and reduce the impact of long-term symptoms.

The National Neuroscience Centre is also involved in trials for nervous system diseases, including amyotrophic lateral sclerosis linked to FUS mutations, with the goal of assessing whether emerging treatments can support clinical function and survival.

• Studying FUS-ALS and its effect on movement and daily function
• Evaluating a potential therapy for motor neuron disease
• Exploring ways to improve outcomes in a serious neurological disorder

These studies highlight the site’s focus on difficult-to-treat neurological conditions, where clinical research is aimed at finding better options for patients facing progressive disease.

In the area of psychiatry and psychology, the site is running research on cocaine addiction, examining whether psilocybin with psychological support may be a useful approach for cocaine use disorder.

• Assessing feasibility of a new treatment approach for cocaine use disorder
• Studying the role of psychological support alongside a novel therapy
• Informing future trials for substance use disorders

This research is designed to help determine whether innovative mental health interventions can be developed into larger studies and, eventually, more effective care options for people affected by addiction.