Learn more about research methodologies for rare disease clinical trials through the lens of rare metabolic conditions in children & rare eye conditions in adults:
Efficacy and safety of a synthetic biotic for treatment of phenylketonuria: a phase 2 clinical trial Dr Kevin Gaughan, Paediatric Specialist Registrar, Children’s Health Ireland
Stickler Syndrome: an overview of the NHS care pathway
Dr Bridget Moran, Ophthalmology Senior House Officer, Mater Misericordiae University Hospital
Moderator: Dr Adam James, Children's Health Ireland