• Medical Science

    Medical Science Recognizes Almost

    7,000 Rare Disorders

    (Jessica’s parents are most
    concerned about one.)

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    Finding answers begins with the

    Building Blocks of life

    (See why identifying changes in DNA is the key.)

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  • Modes of Inheritance

    Finding answers begins with the

    Modes of Inheritance

    (These videos explain genetic disorders in children)"

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When disease gets this personal, so do we.

Rare diseases. Some may not have names, but every child who suffers from one does. The causes are usually unique to each child, and are often found in their genetic makeup. Our mission is a united effort – not to find “one cure” for many, but to help the many children and their families find the “one answer” for them. 


You want answers. So do we. It’s why we begin with research – research that leverages today’s technology to help us identify the underlying causes that lead to disease.

See why it begins with DNA > Learn More

Every Child Matters

Even among the rare diseases that have been recognized by medical science, most are so rare that they are usually overlooked or undiagnosed. We believe whether it’s known or unknown, disease is still disease and one child suffering is one child too many.

Learn More 

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Research & Treatment

Our team of experts and faculty work hand-in-hand, using laboratory research and clinical data to turn key findings into potentially effective therapies and treatments tailored to each child. 

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