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My friend Hakon … is one of the leading geneticists in the world. This book is about one of his most important discoveries—the genetic cause of ADHD, autism and 22q and a potential treatment for these three diseases.

Dr. D. H. Moskowitz

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About the book

This book is about a neighborhood drug company that consisted of three couples. They joined together to help develop a drug that could reverse the consequences of the genetic problems causing ADHD, autism and a rare disease called 22q, for a substantial portion of the children who suffer from these three diseases. With the help from several other people on Dr. Hakonarson’s team, they were able to complete a clinical trial to prove that their drug would be safe and effective in treating the children who had the genetic defect.

This accomplishment is a prime example of President Obama's Precision Medicine Initiative because it provides a therapy based upon genetics that targets subgroups of the population that share the same mutation, even though they have different diseases. The discovery described in this book could improve the lives of millions worldwide.

The discovery was possible because the Children's Hospital of Philadelphia hired Dr. Hakonarson to establish a genetics laboratory and a biobank that collected an enormous number of DNA samples. Research conducted on these samples provided the clue to the genetic defect causing the diseases. The principal scientist, Dr. Hakonarson, found a drug that could reverse the consequences of the mutation causing the diseases.

This book describes how the clinical trial was conducted and the results of the clinical trial. Dr. Hakonarson, with the assistance of the other members of the neighborhood drug company and together with several scientists on his team, was able to provide a proof of concept that the drug would be effective.