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
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…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…Continue
Sharon Hesterlee, Ph.D.
Senior Director Research and Advocacy
Parent Project Muscular Dystrophy…Continue
Making a Drug
By Sharon Hesterlee, Ph.D., Sr Director Research and Advocacy