Assoc ProfessorDeclan O'Rourke

I am a Paediatric Neurologist with interest in advanced therapeutics for children with neuromuscular disorders. As previously indicated I was awarded a grant in 2018 from the Temple Street Childrens Hospital Foundation for a project designed at developing sustainable infrastructure in Neuromuscular Clinical Trials at CHI at Temple Street. My project provided the infrastructural resources to attract and secure neuromuscular clinical trials and sustain the running and patient recruitment of these trials in Children’s Health Ireland at Temple Street. 

I am/have been Principal Investigator in the following clinical trials:

a. A Phase III Double-blind, Randomized, Placebo-Controlled Study assessing the Efficacy, Safety and Tolerability of Idebenone in Patients with Duchenne Muscular Dystrophy Receiving Glucocorticoid Steroids (NCT02814019).

b. A randomised, Double-Blind, Placebo-Controlled, Global Phase 3 study of Edasalonexent in Pediatric Patients with Duchenne Muscular Dystrophy (NCT03703882)

c. An Open-Label Extension Study of Edasalonexent in Pediatric Patients with Duchenne Muscular Dystrophy. (NCT03917719)

d. A Double-Blind, Placebo-Controlled Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy (NCT02500381).

e. A Randomized, Double-Blind, Dose Finding and Comparison Study of the Safety and Efficacy of a High Dose of Eteplirsen, Preceded by an Open-Label Dose Escalation, in Patients With Duchenne Muscular Dystrophy With Deletion Mutations Amenable to Exon 51 Skipping (NCT03992430).

f. Escalating Dose and Randomized, Controlled Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy (NCT04089566).

g. A Prospective, Long-Term Registry of Patients with a Diagnosis of Spinal Muscular Atrophy (SMA) – RESTORE (NCT04174157)

h. Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping (DELIVER) (NCT05524883)

My research team has interests in advanced therapeutics for neuromuscular disorders such as gene modification therapies and gene replacement therapies. My work has involved additional collaborations with the St James’s Hospital-Clinical Research Facility to facilitate gene replacement therapy (onasemnogene abeparvovec) to children in Ireland for the first time, specifically with Spinal Muscular Atrophy (SMA). In 2021, we were the first group in Ireland to administer gene replacement therapy to children in Ireland.

I was appointed Associate Director of Research for Children’s Health Ireland and Deputy Director of the CHI Clinical Research Facility in 2021. My role is to coordinate and grow the advanced therapeutics medicines program at CHI. 

I am a co-applicant on the 2022 CHI-CRF HRB grant, an application for >€5 million designed to ensure the sustainable infrastructure for ongoing clinical trials at CHI.

I have ongoing clinical and research collaborations with the Clinical Research Facility at St. James Hospital.

I am currently supervising an MD Thesis through Trinity College Dublin where my MD student is examining the Prevalence of Paediatric Neuromuscular Disorders in Ireland.

I am currently supervising MD thesis on Quality of Life outcomes in Irish Children with SMA through the UCD school of Medicine.