On August 25th, AVI BioPharma issued a press release stating their planned presentation of the full set of data from a completed phase I clinical trial for drug candidate AVI-4658. As I’ve written before, Charley’s Fund has provided key funding for AVI BioPharma research into finding a therapy via exon-skipping technology, specifically to treat Duchenne Muscular Dystrophy.
Here’s a quote from the release that sums up why so many people are excited about this technology and this type of progress.
“Data from the completed single-blind, placebo-controlled and dose escalationPhase 1 trial showed that AVI-4658 was safe when injected intramuscularly and successfully induced the production of dystrophin protein in patients in a dose-responsive manner. AVI also announced today that these data have been published online in the journal Lancet Neurology and will be featured in the October print edition. This safe and well-tolerated production of new dystrophin is believed to be the key to restoring muscle function and successfully treating patients with DMD – a condition for which there is no currently approved therapy.”
The full press release is available here.
This is the type of research that Charley’s Fund supports. This is the type of research that represents real progress. This is the type of research that can help this generation of boys with DMD. This is also they type of research that requires significant funding to bring these therapies out of trials and into the clinic. We can all help by purchasing the Live Inspired app, donating to Charley’s Fund or Darius Goes West. You can even buy stock in AVI BioPharma (Nasdaq: AVII). Donate time. Share the message. Make sure that your friends and family understand the urgency with which we must approach this cause. Please help.