Who We Are A-T Children's Project

Ataxia-telangiectasia (A-T) is a genetic disease that causes loss of muscle control and balance, cancer, lung disease and immune system problems in children & young adults, shortening their lives. The nonprofit A-T Children’s Project partners with academic and industry investigators worldwide – organizing and supporting innovative research, conferences, clinical teams, data platforms and biomarker development – to optimize disease management strategies, develop new treatments and find a cure.

A Message from the Founders

Anyone whose life is touched by ataxia-telangiectasia is changed forever. Whether the patient diagnosed is your child — or your niece, nephew, grandchild or friend — old assumptions have to be discarded and new realities accepted. The world is suddenly changed, and the future is most uncertain. It is time for making adjustments — physical and psychological — many of them agonizingly difficult.

At the A-T Children’s Project we understand how you feel. When two of our sons were diagnosed, we responded to our personal experience by forming this organization and making a commitment to change that uncertain future, aiming at nothing less than a cure for A-T.

Brad and Vicki Margus

Our Mission

To encourage and support excellent laboratory research which will accelerate the discovery of a cure or possible therapies for ataxia-telangiectasia by:

awarding competitive research grants to top scientists using a peer-review board comprised of top scientists and physicians,
organizing and sponsoring workshops and symposiums in order to encourage cooperation among laboratories and to generate new research strategies, and
working with Congress and the National Institutes of Health to encourage the funding of active research on A-T by agencies of the U.S. government.

And:

To improve the accurate and timely diagnosis of A-T patients by increasing public awareness and by educating physicians.
To develop and maintain an international patient registry of A-T patients with objective, neutral oversight, while leaving ultimate control in the hands of treating physicians, so that up-to-date clinical information about A-T patients can be obtained for researchers and so that when a treatment is developed, all patients can be reached through their physicians.
To support and oversee a clinical center and information clearinghouse at a top-rated, world-class medical center for the evaluation of A-T patients by a multidisciplinary team of specialists, and for the accumulation of experience in managing the many facets of A-T such as ataxia, cancer, and immune problems.
To develop quantitative endpoints for objectively measuring the progression rate and severity of the symptoms of A-T.
To maintain and enlarge a tissue/cell bank with objective, neutral oversight, and control in order to ensure free access of existing and new researchers to A-T patient specimens.