GM1 Gene Therapy Update – April 2015

Dr. Martin of Auburn University and Christine Waggoner, the president and executive director of the Cure GM1 Foundation are pictured.

We recently received updates on the progress of the GM1 gene therapy research.  All of us are extremely eager to get the IND application to the FDA submitted.  As parents,  the clinical trial represents hope.  However, we know time is our children’s enemy.

The reality of the situation is that safety is paramount.  The preparations and application process are very time-consuming.  Furthermore, once the application is approved by the FDA, the drug must be manufactured.  There is a schedule and waiting list for manufacturing.  Steps are being taken to organize the process in the coming months.

The researchers and clinicians want nothing more than to save the lives of children suffering from GM1.  

The funds raised thus far will be used towards next generation techniques.  These newer techniques differ from those of the trial being planned.  The current hope is that the trial may be initiated by the first quarter of 2017.  Progress is being made.  However, there are unknowns and the path to a clinical trial is long.  Continued patience is a virtue.  We must never give up hope.

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