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Jul 28, 2009

U.K. DMD Charity to Provide AVI BioPharma $1.2M to Progress Drugs

  • Action Duchenne has earmarked about $1.2 million for a collaboration focused on progressing AVI BioPharma’s exon-skipping drug candidates for the treatment of duchenne muscular dystrophy (DMD).

    The company reports that its lead candidate, AVI-4658, is designed to skip exon 51 of the dystrophin gene, restoring the reading frame of the mRNA sequence, and resulting in a truncated but potentially functional form of the dystrophin protein. A dose-finding clinical trial evaluating systemic delivery of AVI-4658 is currently under way in the U.K.

    A second, preclinical-stage candidate, AVI-5038, is based on a second generation of AVI BioPharma’s phosphorodiamidate morpholino oligomer antisense chemistry and is deisgned to skip Exon 50.

     



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Scientifically Studying Ecstasy

MDMA (commonly known as the empathogen “ecstasy”) is classified as a Schedule 1 drug, which is reserved for compounds with no accepted medical use and a high abuse potential. Two researchers from Stanford, however, call for a rigorous scientific exploration of MDMA's effects to identify precisely how the drug works, the data from which could be used to develop therapeutic compounds.

Do you agree that ecstasy should be studied for its potential therapeutic benefits?

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