|
New Therapy Save Michigan Boy's Life
JANUARY 14, 2009Jill Harris, Happy News StaffA new therapy to suppress the immune system has helped 2 1/2 year old Ira Brown become the oldest survivor of the CRIM negative form of Pompe disease. Pompe is a rare enzyme disease where in its younger victims causes a loss of a gene necessary to break down glycogen, or stored sugar. As a result, glycogen is built up and damages heart and skeletal muscles. As a defense, many infants inflicted with the CRIM negative form of Pompe disease build up antibodies and rarely live to see their first birthdays. However, the innovative technology led by Dr. Nancy Mendelsohn of Children's Hospitals and Clinics of Minnesota used a novel treatment plan using Rituxan, or rituximab, a drug used for non-Hodgkin's lymphoma and rheumatoid arthritis. This drug allows for the weakening of a child’s immune system response, and has worked in Brown’s case. Mendelsohn is hopeful the treatment will induce tolerance to enzyme-replacement therapy, so the Rituxan may one day be discontinued. To read more about the progress being made by Mendelsohn and her team click here.
|
