At the recent World Muscle Society meeting, which took place in Portugal, a great deal of new data on exon skipping was presented, both on the status of current human clinical trials and on the efforts of investigators to improve the efficiency of the technique by combining exon-skipping with gene and stem cell delivery. Below are a few highlights from the meeting with the caveat that it was impossible to cover every poster and talk. 

 

Muscle Stem Cells

Jenny…

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I’ve just spent two weeks in a row on the road at different meetings—first the Health Research Alliance (HRA) meeting in New York and then the Muscular Dystrophy Association meeting in Las Vegas.

 

HRA is an organization made up of most of the major non-profit funders of health research -- the members meetings are designed to keep us up to date on the latest trends in research and funding and to allow us to learn from one another.  I chair the HRA Working Group on “Funders of…

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I spoke at length last week with Dr. Rhodes about ways that we could better determine the effectiveness for DMD of his VECTTOR electrical stimulation system, which Dr. Rhodes says stimulates acupuncture points, reflexology points and free nerve endings. Dr. Rhodes was very up front about the fact that he isn’t a scientist and isn’t an expert on DMD and his actual professional training is in podiatry. He has supplied his VECTTOR machine to 36 families with muscular dystrophy—he didn’t specify… Continue

There’s often a fine line between “out-of-the–box” and “out-in-left-field.” I guess when my blog about Duchenne research starts with that statement you know I’m probably going to come down one way or another about something, but it might not be the direction you’re expecting.



This community lives with a lot of hope and hype. Many of you have learned that when you hear something too good to be true that it’s probably not. You know the principles of scientific experimentation, you… Continue

The news that GSK is not pursuing a phase III study for skipping exon 51 here in the US has certainly come as a blow. It seems that the FDA would like to see longer term toxicology data before approving the 48 week human study. Meanwhile, almost every European family with DMD will have access to the phase III study. It seems obvious that the FDA is being blindly obstructionist and GSK is showing its true stripes as money-sucking big pharma company that is going to choose the path of least…

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May 7, 2010



I’ve been traveling pretty steadily for more than a week now—first out to Seattle to meet with AVI Biopharma at their headquarters, then to Chicago for the Biotechnology Industry y Organization meeting and finally to Ottawa for the “New Directions in Muscle Biology and Disease” meeting. I thought I would take this time to share with all of you what I’ve seen and heard.



First, it was good to put faces to names at AVI, the Seattle-based biotechnology company that… Continue

Sharon Hesterlee, Ph.D.

Senior Director Research and Advocacy

Parent Project Muscular Dystrophy…

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Making a Drug

By Sharon Hesterlee, Ph.D., Sr Director Research and Advocacy

February 8,…

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