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ea0033oc3.3 | Oral Communications 3 | BSPED2013

Successful treatment of four patients with severe hyperinsulinaemic hypoglycaemia with a novel therapy using mTOR inhibitor

Senniappan Senthil , Alexandrescu Sanda , Tatevian Nina , Shah Pratik , Arya Ved , Flanagan Sarah , Ellard Sian , Rampling Dyanne , Ashworth Michael , Brown Robert , Hussain Khalid

Introduction: Hyperinsulinaemic hypoglycaemia (HH) is the most common cause of severe and persistent hypoglycaemia in neonates. The treatment of diazoxide unresponsive HH involves pancreatectomy. Mammalian target of rapamycin (mTOR) is a protein kinase that regulates cellular proliferation. We aimed to evaluate the efficacy of mTOR inhibitor Sirolimus and assess mTOR expression in the pancreas of infants with severe HH.Methods: Four infants with severe, ...

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Successful treatment of four patients with severe hyperinsulinaemic hypoglycaemia with a novel therapy using mTOR inhibitor

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