Participation in a research study helps to:

• Find treatments for existing diseases to improve the health of both children and adults.
• Identify the best dose of medicines to prevent harmful effects or under-treatment.

It is very important to understand that research is conducted to gain information about a disease, condition, drug, or treatment that will benefit people in the future. Although, your family might not benefit from the results of the study, your participation will certainly help the greater community affected with the condition, including future generations of your family.

There are several benefits to participating in a research study. These include:

• Researchers test new drugs and treatments because they have reason to believe they might work better or be safer than the standard care. In a study, you or your child may have access to a treatment that is not available yet. If the drug or treatment is found to be helpful, you or your child may be among the first to benefit.
• Enrolling in a study may give you or your child a chance to see extra doctors or find out more facts about the medical condition. A study may connect you with other families going through the same medical challenges that you are experiencing.
• A study may offer closer monitoring or additional testing for you or your child, which may not be part of regular care. Sometimes a study asks patients to keep a diary or have more frequent medical exams. Everyone in a study is monitored closely.

Choosing to participate in a research study is an important personal decision. Here is some information to help you make that decision.

The government has strict guidelines and safeguards to protect people who choose to participate in research studies. Each study that uses human subjects must be approved and monitored by the Institutional Review Board (IRB) of the institution where the research is to be conducted. The IRB’s job is to make sure the risks are as low as possible, the studies are worth any potential risks, and that these risks and benefits are explained in a simple and clear manner for the participants to make an informed decision. The Marfan Foundation requires that a study have IRB approval before we tell the Marfan syndrome and related conditions community about it.

Before you can enroll in a study, you will be asked to sign an “informed consent.” “Informed consent” is the process of learning the key facts about a study before you decide whether or not to participate.

These facts include:

• Why the research is being done?
• What the researchers want to accomplish?
• What will be done during the study and for how long?
• What risks are involved in the study?
• What benefits can be expected from the study?
• Can I decline participation in the study at any time?

If you wish to participate in a study, the research staff will give you informed consent documents that describe the study and its guidelines. You may want to discuss the consent documents with your friends and family members. In addition, you should ask the research team any questions you have before you make a decision, during the study, and after the study.

Some other questions you might want to ask are:

• Will I benefit from the results of this study or will others?
• Will I have access to the results of this study?
• Will my samples or records be destroyed or returned in a reasonable amount of time?
• Are there any inconveniences such as expenses, time, and medications associated with the study?
• Who will have access to my medical information?
• Is this study suitable for children?
• Is there a written guarantee of privacy?
• Will there be pain – either physical or emotional – associated with the study?
• Who is sponsoring the study?
• Is this study repetitive?

Plan ahead and write down the questions you want to ask. You should not incur any expenses due to participation in a study. Participating in a research study does not entitle you to free medical care from the research investigators unless explicitly stated in the consent form. If you decide to participate, always obtain a copy of your signed consent form.