New Clinical Trial

A representative of Kennedy Krieger Institute has recently informed me of a new clinical trial for boys with Duchenne Muscular Dystrophy. They are checking to see if the drug Revatio (same drug that is in Viagra) would be beneficial as a cardiac drug for DMD patients. They are still recruiting for this trial. They plan to have 30 boys, ages 15 or older, who have been diagnosed with DMD. For more information, or to see if your son qualifies, click here.

I have recently read about the potential benefit of this drug in boys with DMD in a Quest article. I am so thankful for so many researchers trying to help these boys!


About the Kennedy Krieger Institute
Internationally recognized for improving the lives of children and adolescents with disorders and injuries of the brain and spinal cord, the Kennedy Krieger Institute in Baltimore, MD serves more than 16,000 individuals each year through inpatient and outpatient clinics, home and community services and school‐based programs. Kennedy Krieger provides a wide range of services for children with developmental concerns mild to severe, and is home to a team of investigators who are contributing to the understanding of how disorders develop while pioneering new interventions and earlier diagnosis. For more information on Kennedy Krieger Institute, visit www.kennedykrieger.org.

Clinic October 2010

We had another good visit with Dr. Flanigan today. There is encouraging news coming with these exon skipping trials. Currently they are doing a trial skipping exon 51. (They are still enrolling for this! For more information, click here). So far, so good. If exon 51 goes well and is tolerated, then they will move to exon 44, and after that exon 45 (the one that Ben could benefit from).

He does want Ben to have another echocardiogram. He didn't say why but it looks like we will do that in the near future. His steroid dosage will be upped from 15 to 18mg. Overall, he said that Ben appears to be a less severe DMD boy. That is VERY good news to me. :o)

Here is another link to Dr. Flanigan's podcast. It features authors of publications of recent studies.