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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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