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Frequently Asked Questions

The DRAGONFLY Study

Thank you for your interest in DRAGONFLY Study. Please see below some frequently asked questions. A full list of frequently asked questions can be viewed here. If you have additional questions that are not listed, please contact us!

What is the purpose of the DRAGONFLY Study?

The purpose of the DRAGONFLY Study is to bring the SCN2A-Related Disorder community together and collect data.
Some of the goals of the DRAGONFLY Study are:

  • To describe the people who have SCN2A-related disorders and to better understand the stages of the disease and the different ways the disease affects people. To do this, we will ask about diagnosis, treatment, medical history, social and economic environment, and treatment outcomes.
  • To understand how SCN2A-related disorders change over a person’s lifetime and to learn about clinical practice patterns and variations over the course of treatment.
  • To help to develop best practices, management guidelines and recommendations so that clinicians can know how to give the best care to improve the quality of life and outcomes of people with SCN2A-related disorders.
  • To identify people with SCN2A-related disorders who might be willing to take part in other research studies or clinical trials. You will be able to choose whether you want to hear about these other studies.

What is a Patient Registry?

A patient registry is a collection of standardized information about a group of patients who share a condition. The information may be used for a variety of purposes such as conducting natural history studies and supporting disease specific clinical trial recruitment.

What is a Natural History Study?

A natural history study is a study designed to track the course of a disease over time. It includes people who have a specific medical condition or disease. It may also include those who are at risk of developing the condition/disease. This type of research identifies demographic, genetic, environmental and other information that may be common within the disease and its outcomes. A natural history study can also show the differences in symptoms and changes over time that are seen in different people with the same disease. Natural history studies often aim to find unknown similarities within the disease population. They have many potential uses such as patient care best practice development and clinical trial recruitment. Data for natural history studies are often collected via patient registries.

How is the data collected?

Data is collected through a secure web-based application (that can be accessed by computer, tablet or phone) developed by the National Organization for Rare Disorders, Inc. (NORD®), (learn more about NORD below). Study participants respond to questions grouped within a series of surveys developed per study standards and in collaboration with disease specific experts.

What types of data will be collected in the DRAGONFLY Study?

The data collected includes but is not limited to:

  • Socio-demographics
  • Medical and diagnostics
  • Treatment and disease progression
  • Management of care
  • Quality of life

Who is FamilieSCN2A Foundation?

FamilieSCN2A Foundation is a nonprofit organization created by parents of children diagnosed with rare forms of epilepsy and autism as a result of a change in the SCN2A gene. Since its founding in 2015, FamilieSCN2A Foundation is working to build a world with effective treatments and cures for all SCN2A-related disorders. Its mission is to accelerate research, build community, and advocate to improve the lives of those affected by SCN2A-related disorders around the world. With unwavering dedication, the foundation provides a nurturing and informative community where clinicians, scientists, parents, caregivers, and individuals with SCN2A-related disorders come together to share experiences, knowledge, and hope. Visit scn2a.org.

Who can join the study?

This study is open to anyone who has a SCN2A-Related Disorder diagnosis and meets the study inclusion criteria for participation.

Is there a cost to participate?

There is no cost to the patient to join this study.

How long will this study last?

A registry on the IAMRARE platform will typically be open for at least five years. Participants will be asked to return to the registry periodically to update their information.

Can data be collected worldwide?

The registry uses an online platform which allows participants to contribute data from anywhere in the world. Individuals from other countries who enter data into the registry should be aware that data and privacy laws are different in the U.S. from other countries. This U.S. based registry will protect data and privacy according to U.S. requirements.

Who is NORD – the National Organization for Rare Disorders, Inc.?

NORD, an independent nonprofit, is leading the fight to improve the lives of rare disease patients and families. NORD does this by supporting the rare community, its people, and organizations. NORD works together to accelerate research, raise awareness, provide valuable information, and drive public policy that benefits the estimated 25-30 million Americans impacted by rare diseases.
Learn more about NORD at https://rarediseases.org/.