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.