A $200,000 matching donation was offered at our October gala. For the months of October, November and December 2008, each dollar that you donate to CureDuchenne, up to $200,000, will be matched by an anonymous donor. We thank this person for this support, and we ask you boldly to help us maximize this contribution. Write "Paddle" on your check dated before December 31st, and it will be matched.

and James Tupper Willie Garson

PTC THERAPEUTICS
PTC has two DMD projects underway. Through another organization, Parent Project MD, CureDuchenne contributed substantial funds towards the initiation of these projects.

PTC124
This drug acts to ignore the defect in the gene, allowing the gene to produce dystrophin, the missing protein responsible for Duchenne. This drug is in a phase 2 trial in many DMD boys. The results will not be formally announced until next year, but the anecdotal evidence from some parents is encouraging. Even though PTC124 will only treat about 10% of DMD boys with a particular type of mutation, we are all very excited to see these boys tolerate the presence of dystrophin in their bodies and this gives us all reason to believe that Duchenne can be conquered.

PTC Small Molecules
PTC is developing drugs that will increase or decrease other proteins in DMD boys that can help compensate for the lack of dystrophin. They continue to move closer to an actual drug which can move through the testing stage and hopefully into human trials.

PROSENSA
It's been five years since we agreed
to support Prosensa's exon skipping project. Exon skipping tricks the defective gene to produce the dystrophin protein that is missing and the cause of Duchenne muscular dystrophy. We are so happy to announce that they are currently in human trials in actual DMD patients. Although they won't be able to announce the results until early next year, we are encouraged that there have been no adverse reactions...a very good thing for a
completely new kind of drug.

NON-HORMONAL STEROIDS
Corticosteroids are the only available treatment we have now for Duchenne. They have improved the quality of life for many of our children, but they carry many serious side effects. Parents of DMD boys have dreamed of having a therapy that could provide the benefit of steroids without the awful side effects. CureDuchenne partnered with an Italian team of scientists who are now testing their drug in human DMD patients. This won't be a cure but we are hopeful that we can relieve our children of some of the dreaded effects of chronic and large dose steroids.

ADULT STEM CELLS
Dr. Giulio Cossu has identified what seem to be the most promising adult stem cells to use for Duchenne research. CureDuchenne funded Dr. Cossu last year to enable this
project to move forward by expanding the cell lines. His group is currently working on a one year program to validate the outcome measures of a #human clinical trial.

CureDuchenne is a relatively small organization, but we have been very effective in leveraging our donor's dollars into large grants and investments from venture capital firms and government institutions. Our goal is to provide scientists the funding they need to take a project that has achieved proof of concept to a pre-clinical or clinical trial. If successful, we know that larger organizations will provide the large amount of funding necessary to take the technology or drug to human trials.