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NIH TRND program announces next round of drug development projects
Date:11/15/2011

at afflicts more than 200 million people worldwide. The disease can cause severe anemia, diarrhea, internal bleeding and/or organ damage. Neglected diseases are conditions that inflict severe health burdens on the world's poorest people. This project aims to produce compounds that retain the positive anti-parasitic effects of the current treatment, praziquantel, which stuns and kills the worms while enabling lower and less frequent doses with potential for improved tolerability. This may allow broader access of a therapeutic to affected patients.

A drug candidate for treatment of Duchenne muscular dystrophy.
Peter Sazani, Ph.D., Executive Director of Preclinical Development
AVI BioPharma, Bothell, Wash.

Duchenne muscular dystrophy is an inherited, rapidly progressive form of muscular dystrophy affecting approximately 1 in 3,500 male births worldwide. This collaboration aims to develop a compound that would treat a sub-group of patients with a specific mutation responsible for Duchenne muscular dystrophy. The team will also investigate the general utility of this innovative treatment platform technology.

A pharmacological therapy for treatment of autoimmune pulmonary alveolar proteinosis.
Bruce C. Trapnell, M.D., Francis R. Luther Professor of Medicine and Pediatrics
Cincinnati Children's Hospital Medical Center-Research Foundation, Cincinnati, Ohio.

Pulmonary alveolar proteinosis is a rare lung disease characterized by the build-up of a grainy material in the air sacs of the lungs that causes breathing difficulties and can result in respiratory failure in rare cases. The protein therapeutic that is the subject of this collaboration will be developed as an inhaled therapy.

TRND has established data-driven milestones for each project to track progress and allow projects which do not achieve milestones in the established timeframe to be terminated, thus allowing other promising candid
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Contact: Geoffrey Spencer
spencerg@mail.nih.gov
301-451-8325
NIH/National Human Genome Research Institute
Source:Eurekalert

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