Thank you for your interest in our survey.

We are asking people directly affected by, or involved with, Batten disease (neuronal ceroid lipofuscinoses, NCLs) to tell us what research questions they feel are the greatest priority for Batten disease research. These may be broad questions or very specific. The purpose of this study is to determine unanswered research questions that should be prioritized by researchers and research funders. This will ensure that future research is directed towards the areas that matter most to people in the Batten disease community. The responses you provide may play an important role in helping to guide and shape future Batten disease research.

This project has two phases. The first phase is an open, free-response survey. We will collect research questions from stakeholders across the Batten Disease community (Survey 1). We will categorize all of the questions received to produce a summary list of research questions. In the second phase, we will ask stakeholders to rank those research questions in order of importance (Survey 2). We will use those rankings to determine the top priority research questions identified by the Batten disease community.

You may participate in Survey 1 only, Survey 2 only, or both surveys. Right now, we are collecting information for Survey 1.

The survey should take around 10-15 minutes to complete. We will ask questions about your background, such as your gender, race, ethnicity, age, geographical location, and how you are associated with the Batten disease community. We will ask you to share the Batten disease-related research questions that are important to you. We anticipate up to 500 people will take part in this project.

Completing this survey is completely voluntary (it is up to you). If you begin the survey and change your mind, you may stop at any point.

Who should answer the survey questions?
We invite you to complete this survey if you are aged 18 years or over and are a part of any of the following groups:
• You are a person with a Batten disease (NCL) diagnosis
• You are a parent/caregiver of a person with a Batten disease (NCL) diagnosis
• You are a professional involved in diagnosing or treating people with a Batten disease (NCL) diagnosis
• You are a professional involved in Batten disease (NCL) research
• You are a professional involved in Batten disease (NCL) drug development or drug approval regulation
• You are involved in advocacy for people with a Batten disease (NCL) diagnosis or for Batten disease (NCL) research
• You are involved in another way with people with a Batten disease (NCL) diagnosis (support worker, educator, relative, friend, other)

What will happen to my question(s) or the information I share?

Each submitted question or topic will be reviewed. If a question has already been answered by research and published, it will be excluded. Questions that have not been answered by prior research will compiled into a second survey (Survey 2). In the second survey, the Batten disease community will be asked to rank research questions in order of importance.

By participating in this survey, you give us permission to publish the research questions you propose. The surveys are anonymous; no one will know that you (or anyone else) were part of the study.

This survey is being led by Dr. Erika Augustine at the Kennedy Krieger Institute in partnership with the Batten Disease Support, Research, & Advocacy Foundation (BDSRA). If you have questions about this survey, please contact Dr. Augustine or Nadia Moore at batten@kennedykrieger.org