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Orphanet Journal of Rare Diseases
Published in: Orphanet Journal of Rare Diseases 1/2017

Open Access 01-12-2017 | Letter to the Editor

Extreme caution on the use of sirolimus for the congenital hyperinsulinism in infancy patient

Authors: Indraneel Banerjee, Diva De Leon, Mark J. Dunne

Published in: Orphanet Journal of Rare Diseases | Issue 1/2017

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Abstract

We have recently published on the limited effectiveness of sirolimus as a treatment option for hypoglycaemia as a consequence of hyperinsulinism. Our data oppose the view that mTOR inhibitors provide new opportunities for the treatment of patients with hyperinsulinism. We are not convinced by the argument that any benefit for some patients outweighs the potential and later long-term problems that accompany mTOR inhibition in the neonate. We also express the opinion that caution must be taken when repurposing/repositioning therapies in the field of rare disease.
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Metadata
Title
Extreme caution on the use of sirolimus for the congenital hyperinsulinism in infancy patient
Authors
Indraneel Banerjee
Diva De Leon
Mark J. Dunne
Publication date
01-12-2017
Publisher
BioMed Central
Published in
Orphanet Journal of Rare Diseases / Issue 1/2017
Electronic ISSN: 1750-1172
DOI
https://doi.org/10.1186/s13023-017-0621-5

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