Pre-clinical development of a novel oral therapy for hyperinsulinemic hypoglycaemia

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Congenital Hyperinsulinism is a rare neonatal disease presenting severe hypoglycaemia associated with poor clinical outcomes and long-term neurodisability.
The support of the Technology Strategy Board will enable Heptares, in collaboration with the University of Manchester and Great Ormond Street Hospital, to carry out the pre-clinical development of a novel safe, effective and orally available treatment for Congenital Hyperinsulinsm. A successful outcome will provide a clinically ready molecule with anticipated additional benefits in other areas associated with intractable hypoglycaemia; including neonatal hypoglycaemia, hypoglycaemia occurring as a consequence of gastric bypass surgery and insulinoma-associated hypoglycaemia.