ROADMAP Tutorial Video

If you would like to find out more about how best to navigate the ROADMAP tool and survey insights pages, watch the short tutorial below, by ROADMAP project lead Ania Korsunska.

State of rare disease nonprofits in the US

As our first step to building the ROADMAP, we set out to build a comprehensive list of all rare disease nonprofit organizations in the US. A team of volunteers spent seven months extracting data from the websites of these organizations, gathering basic information such as their year of founding, name & contact information of founders, information on the organization’s resources (conference, research agenda, biobank, registry, natural history study, etc.), whether the rare disease has treatment guidelines, or whether it is known that it is caused by a genetic mutation, is there any mention of drug repurposing, and if so - which drug(s) were pursued, or is there any mention of partnerships or collaborations with other organizations. As a result of this exercise, 711 organizations were confirmed as rare disease nonprofit organizations in the US with active websites as of 12/2021. Because this data is publicly available through organizations' websites and separate from the ROADMAP survey, we have made available open-source here. Click the link below to download the file.


PDFs of the ROADMAP content

For your convenience, we also are providing the following in PDF form if you have trouble accessing the information on the interactive tool: Click the link below to download the files.

About the ROADMAP Project:


ROADMAP tool content:


ROADMAP Paper [coming soon]

A paper describing the findings from this project will be submitted to a peer-review, open source journal soon, and will be made available for download here as soon as possible.

In the meantime, if you would like cite this project or the data available in the data explorer, please utilize this citation: Korsunska, A., Bolden, S. E., Repasky, M., Zuccato, M., Fajgenbaum, D. (2023) The ROADMAP Project.