AZ, Bristol report promising results in study of drug targeted rare disease

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AZ replace othersBristol-Myers Squibb Company and AstraZeneca reported promising results from a 12-month analysis of a National Institutes of Health long-term research study of metreleptin.

The drug is targeted at lipodystrophy a rare disease estimated to affect a few thousand people around the world, often at an early age. Thirty-nine patients were involved.

The full study was initiated in 2000 by investigators at the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), part of the NIH, and is currently ongoing.

We are encouraged by the results of this longer-term analysis,” said Jean L. Chan, M.D., Medical Director, Bristol-Myers Squibb. “Bristol-Myers Squibb and AstraZeneca recognize the unmet medical need of pediatric patients affected by lipodystrophy and the importance of studying potential treatments for this rare disease.”

Patients with lipodystrophy experience loss of fat tissue, especially fat under the skin. Without enough fat tissue or leptin, the body’s system for regulating energy use and storage falls out of balance and leads to various complications. AstraZenea has administrative and manufacturing operations in northern Delaware. 

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