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UPDATE ON DRUG TRIAL IN THE USA FOR JUVENILE BATTEN DISEASE

You may have seen on the BDSRA’s website that Dr. Pearce and the group at the Batten Center, Directed by Jon Mink, at the University of Rochester Medical Center are preparing for a human trial of the drug, Mycophenolate Mofetil (CellCept) to determine if it has any affect on the progression of the disease. This is based on information that CellCept does have a positive effect on some aspects of disease progression in CLN3 mice. More work is still needed to find out exactly what is happening in the CLN3 mice but the big question will be whether CellCept can do the same thing in children/young adults with Juvenile Batten Disease? The BDSRA want to raise $500,000 through their "Make the Juvenile Trial Happen” campaign to find out.  For more information see their website at: www.bdsra.org.

The U.S. Food and Drug Administration (FDA) has just approved the drug for a US trial and the next step is for them to obtain Institutional Review Board (IRB) approval from the university. We are in active contact with Dr Jon Mink and Lance Johnston, Executive Director at the BDSRA, as to their progress in setting up the Clinical Trial with a view to exploring the options for UK children/young adults’ future participation in the trial. When the initial phase begins only US patients will be accepted but we will keep members informed of any developments that occur.

 



 

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