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Pioneering research into rare neonatal diseases
Date:8/13/2012

Groundbreaking research into rare neonatal diseases, conducted by the University of Sheffield, is set to improve the treatment of babies who lack the stress hormone cortisol.

A European Union grant, awarded to the University of Sheffield and Sheffield Teaching Hospitals NHS Foundation Trust with pharmaceutical Diurnal Ltd and partners, will assist the development of a novel neonatal formulation of hydrocortisone a steroid drug that is essential for the survival of babies who lack the hormone cortisol.

Known as the stress hormone, cortisol is secreted from the adrenal gland in the body - without it infants cannot survive. Cortisol has a critical role in combating infections or other sources of physical stress such as extreme exercise or surgery.

The most common cause of adrenal insufficiency in neonates is congenital adrenal hyperplasia (CAH). Approximately 1 in every 12,000 children is born with CAH which is a rare genetic disorder that blocks the adrenal glands' ability to synthesise cortisol.

Due to numerous ethical and practical challenges of testing medications, newborns, neonates and infants are often thought to be at a disadvantage when it comes to new medical treatment, because they have to be treated with drugs that were developed for adults.

Professor Richard Ross from the University of Sheffield's Medical School said: "Adrenal insufficiency is a serious condition resulting from a lack of stress hormone cortisol, and untreated patients will die from an adrenal crisis.

"Currently there is no licensed preparation to treat neonates and infants with adrenal insufficiency, and parents have to use crushed adult tablets, which have associated risks of over- and under-treatment. Under-treatment can be potentially lethal, while over-treatment results in growth failure and obesity."

The European collaborative project Treatment of Adrenal Insufficiency in Neonates and Infants (TAIN) Development
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Contact: Amy Pullan
a.l.pullan@sheffield.ac.uk
44-114-222-9859
University of Sheffield
Source:Eurekalert

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