All parents hope for a long and healthy life for their child. But when your child has WAGR syndrome, that hope can feel like a far-off dream. There is a way to make the dream real. Research is the key to better, healthier lives for children and adults with WAGR syndrome. 

Learn more about medical research, studies involving WAGR syndrome, and how you can help turn hope into reality.

Caroline (adult with WAGR syndrome) standing on HOPE at St Jude Children's Research Hospital

• "Basic science" research improves our understanding of nature. An example of basic science research in Aniridia is the breeding of frogs with this condition, to better understand how aniridic eyes develop before and after birth. Another example is the Tooth Study, a project to examine whether neurons can be grown from the baby tooth pulp of children WAGR syndrome to better understand the brain and behavior
• A "natural history study" is a project that follows a group of people with a particular diagnosis over time to understand more about the condition. The WAGR Syndrome Patient Registry is a natural history study of patients WAGR syndrome

• "Pre-clinical research" involves applying basic science principles to test a hypothesis or prediction. An example of pre-clinical research is the study of a medication to prevent Wilms tumor in mice with a genetic risk for Wilms tumor similar to WAGR syndrome
• A "clinical trial" is a type of research that evaluates a medical, surgical or behavioral treatment. Clinical trials compare a new medication or medical approach to a standard one, or to a placebo (a harmless pill), or to no intervention if no treatment for the condition currently exists. At present there are no clinical trials specifically for patients with WAGR syndrome, but there is hope for these in the future

• A desire to help doctors learn more about diagnosis and treatment of WAGR syndrome and the conditions associated with it

• A desire to help others with this disorder
• Hope that your child will benefit from a particular study or will benefit from it in the future

• The purpose of the research
• An outline of what will take place during the research
• Whether there is any compensation for participating in the research

• What risks are involved 
• What benefits may occur
• How information will be kept confidential
• Who to contact with questions

Different types of studies have varying degrees of risk. Any risks involved in a study will have been identified prior to the start of the study, and will be disclosed to the participants (see Informed Consent). All research studies that involve humans are required to undergo rigorous review by an Institutional Review Board (IRB) before the study can take place. 

IRBs are made up of groups of people (scientists and non-scientists) whose job is to protect the rights and welfare of research participants and to assess risk of a particular study. Vulnerable populations, such as children or people with intellectual or developmental disabilities, are especially well protected. Prior to and during research, IRBs ensure that the study is adhering to ethical norms and that participants rights are protected.

First and foremost, people who take part in research are volunteers. At any point during a research project, a person can decide to quit the study. Study participants also have other rights.