Community Discussion Forum New to PKU cure? Reply To: PKU cure?

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I’m responding quite well, and happy about that. As to the original post asking if Kuvan is a cure – it absolutely is not. It’s a treatment, and not a treatment every PKUer can benefit from. The only possibility I’m aware of for a cure would be if gene therapy could be perfected. Failing that, we’re left with treatments. There is currently a clinical trial for PEG-PAL, which is essentially enzyme replacement therapy. If PEG-PAL proves to be safe and effective (animal testing has shown promise) – it is possible that PKUers could eliminate all dietary restrictions. It would require injections every week or two, if I recall correctly, but if it is effective, it would work for ALL PKUers, including those who do not respond to Kuvan.

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