Principal Investigator is Ching Wang, M.D., Ph.D., a pediatric neurologist at Lucile Packard Children’s Hospital at Stanford University Medical Center. Dr. Wang has conducted research in SMA for more than 15 years and is currently working on finding an effective treatment for SMA. Stanford is the only medical center in the world that is actively studying the use of hydroxyurea in children with SMA. There are currently two ongoing studies: one for Type 1 SMA patients and a second for Type 2 and Type 3 SMA patients. Stanford’s studies were approved by the U.S. Food and Drug Administration and are scientifically sound. Dr. Wang and the research team have years of experience with SMA. Dr. Wang’s research lab uses state of the art technology in genetic and molecular analysis for these clinical trials. The studies are divided into two stages. In the first stage, patients undergo six months of treatment with either hydroxyurea or a placebo under randomized, double-blind conditions. After patients complete six months of randomized treatment, they proceed into the second stage of the study, which uses an open-label design, meaning all participants are known to be receiving hydroxyurea from that point forward. Throughout the study, participants have regular clinic visits with Dr. Wang, regular lab work to monitor both the safety and effectiveness of the treatment, and certain other tests to monitor their muscle function. We have seen interesting clinical improvements in some patients in both stages of the study, but since our study is still in progress we cannot be sure that these improvements are due to the drug treatment. We can only make that conclusion when we are able to systematically analyze the data at the end of these studies. Dr. Wang is the chair of an international standard of care committee for SMA. This committee consists of experts highly experienced in caring for children with SMA. They drafted a statement on the standard of clinical care for children with SMA. Establishing the standard of care for SMA will facilitate the valid measurement of clinical outcomes during clinical trials in the future. Federal research funding has become much more difficult to obtain. Applying to the public funding agencies requires substantial amount of energy and time. This takes away time and energy from the researchers who would like to spend time on the SMA research. Family supports like yours will help to ensure the continuation of our clinical trials and allow us to identify effective treatment rapidly. Any questions can be directed to Dr. Wang at (650) 724-7973 or to his Research Coordinator, Virginia Wedell, at (650) 498-7658.
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