What is a rare disorder?
The U.S. Food and Drug Administration defines a rare disease as one that affects fewer than 200,000 Americans at any given time. Rare childhood disorders are estimated to impact 1 in every 150 births.
What is the mission of the Center for Rare Childhood Disorders?
In order to help affected children and their families, TGen has established the Center for Rare Childhood Disorders (C4RCD). TGen's C4RCD is committed to developing, refining, and applying the latest tools of genomic medicine for the diagnosis and clinical management of patients.
C4RCD's mission is to better diagnose, treat and ultimately cure rare childhood genetic disorders in the 21st Century with a goal of improving the lives of children and families affected by rare disorders world-wide. The C4RCD will use the most advanced technology and analytical approaches to identify a genomic solution that could find the cause of a child's rare disorder and bring new health and hope to countless ill children and adults around the world.
Does the Center need financial support?
Yes. Donations to support our work with children are needed and greatly appreciated. TGen is a nonprofit 501(c)(3) organization, so donations are tax deductible. To make an online donation click here, or call the TGen Foundation at 855-343-8611 for other giving options.
Can I pay TGen to have my child's genome sequenced?
TGen is a research institute and our genome sequencing is performed as part of our research studies. We do not offer it as a service the public can pay for.
Can my child participate in research at the Center for Rare Childhood Disorders?
We are currently enrolling a limited number of participants into our research program. If you would like to be considered for a study, please complete the online form to have your contact information entered into our Research Registry. To learn more about the registry, click here.
What studies do you currently have open?
The current study available is titled, "Genetic Studies of Patients and their Families with Neurological Diseases of Unknown Etiology". This study aims to use molecular technology to identify or further explain genetic contributions to neurological disorders that physicians have not been able to diagnose in children and young adults.
What does "unknown etiology" mean?
Unknown etiology means that the cause of the disease or condition is not known. Unfortunately, children with a known genetic cause for their condition (such as Fragile X syndrome) are not eligible for our current study.
What are biospecimens?
Biospecimens are samples or materials that are taken from the human body. This may include blood, saliva, tissues, tumors, urine or other body fluids. Biospecimens are used in molecular research to better understand the genetics or biology of a disease, condition or drug treatment.
Do we have to be in Arizona to be in the study?
No. Our experienced staff can consent study participants over the telephone and work with your physician to collect biospecimens.
My child does not qualify for this study. Is there another study I can enroll him/her in?
TGen may not currently have an open study for your child to participate in.
Another option is to look at the website ClinicalTrials.gov for other investigators who may be doing research on your child's disorder. This site lists all studies receiving funding from the U.S. government. Patient organizations are also a good source of information on clinical trials.
If you have questions about a particular study, the C4RCD or TGen, please contact us. We will contact you within a week.
Who is TGen?
The Translational Genomics Research Institute (TGen) is a non-profit 501(c)(3) organization focused on developing earlier diagnostics and smarter treatments.
What is translational research?
Translational genomics research is a relatively new field employing innovative advances arising from the Human Genome Project and applying them to the development of diagnostics, prognostics and therapies for cancer, neurological disorders, diabetes and other complex diseases.