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
|Contact: Geoffrey Spencer|
NIH/National Human Genome Research Institute