Company lists · Data refreshed September 2026
Top 25 Rare disease nonprofits based in the United States
RevenueBase identified 210 rare disease nonprofits headquartered in the United States as of September 2026 and tracks 2,108 professional contacts across them, including 781 with verified work emails. Washington is the largest hub.
This list covers rare disease nonprofits headquartered in the United States, matched on what each company actually does as described in its own words. Ranked by team size, largest first. Every row shows the company's own description, its RevenueBase Smart Search rating, and when it was last checked. Data refreshed September 21, 2026.
Published by RevenueBase, a B2B data infrastructure company. This list was built with RevenueBase Smart Search, the natural-language company search RevenueBase customers use to build their own targeted lists and unlock verified emails and direct dials at the companies on them. How these lists are built and judged.
The 25 largest rare disease nonprofits based in the United States
Ranked by team size| # | Company | Contacts | |||
|---|---|---|---|---|---|
| 1 | 185 contacts → | ||||
Indirect evidence Source: RevenueBase Database · checked Sep 21, 2026Thanks. The Data Team will review it. | |||||
| 2 | 168 contacts → | ||||
Strong evidence “Bionews is a digital health solutions company dedicated to empowering more than 50 rare disease communities with trusted information, news, and connections.” Source: RevenueBase Database · checked Sep 21, 2026Thanks. The Data Team will review it. | |||||
| 3 | 156 contacts → | ||||
Strong evidence “The National Organization for Rare Disorders (NORD), a 501(c)(3) organization, is an independent patient advocacy organization dedicated to helping individuals with rare diseases…” Source: RevenueBase Database · checked Sep 21, 2026Thanks. The Data Team will review it. | |||||
| 4 | 81 contacts → | ||||
Strong evidence “The EveryLife Foundation for Rare Diseases is powered by the rare disease community to improve health outcomes by driving change through evidence-based policy, leading…” Source: RevenueBase Database · checked Sep 21, 2026Thanks. The Data Team will review it. | |||||
| 5 | 67 contacts → | ||||
Indirect evidence “Leading with purpose.” Source: RevenueBase Database · checked Sep 21, 2026Thanks. The Data Team will review it. | |||||
| 6 | 63 contacts → | ||||
Partial evidence “This is a time of great hope for individuals with complex diseases such as NF1 and their families.” Source: RevenueBase Database · checked Sep 21, 2026Thanks. The Data Team will review it. | |||||
| 7 | 48 contacts → | ||||
Strong evidence “Uplifting Athletes, a nonprofit organization founded in 2007, harnesses the power of sport to build a community that invests in the lives of people impacted by rare diseases.” Source: RevenueBase Database · checked Sep 21, 2026Thanks. The Data Team will review it. | |||||
| 8 | 46 contacts → | ||||
Strong evidence “Accessia Health is a national nonprofit 501(c)(3) organization that provides programs and services to those living with rare or chronic health conditions.” Source: RevenueBase Database · checked Sep 21, 2026Thanks. The Data Team will review it. | |||||
| 9 | 39 contacts → | ||||
Strong evidence “Empowering the Next Generation Rare Disease Advocate.” Source: RevenueBase Database · checked Sep 21, 2026Thanks. The Data Team will review it. | |||||
| 10 | 36 contacts → | ||||
Strong evidence “TPIRC is a non-profit clinical care and research center that focuses on the development of cutting-edge, individualized treatment protocols for rare and orphan diseases utilizing…” Source: RevenueBase Database · checked Sep 21, 2026Thanks. The Data Team will review it. | |||||
| 11 | 31 contacts → | ||||
Strong evidence “Rare Genomics Institute (RG) was founded in 2011 to fill the health care gap for undiagnosed rare disease patients and supporting research in rare diseases.” Source: RevenueBase Database · checked Sep 21, 2026Thanks. The Data Team will review it. | |||||
| 12 | 28 contacts → | ||||
Partial evidence “New advanced medicines are curing patients of serious diseases.” Source: RevenueBase Database · checked Sep 21, 2026Thanks. The Data Team will review it. | |||||
| 13 | 21 contacts → | ||||
Strong evidence “Indo US Organization for rare diseases (IndoUSrare) is an independent non-profit organization focused on helping patients with rare diseases of Indian origin in the USA, India, &…” Source: RevenueBase Database · checked Sep 21, 2026Thanks. The Data Team will review it. | |||||
| 14 | 19 contacts → | ||||
Strong evidence “Probably Genetic helps undiagnosed rare disease patients find answers to their symptoms in a matter of weeks.” Source: RevenueBase Database · checked Sep 21, 2026Thanks. The Data Team will review it. | |||||
| 15 | 19 contacts → | ||||
Partial evidence “The Isabella Santos Foundation (ISF), a 501(c)3, is dedicated to fueling change in rare pediatric cancer.” Source: RevenueBase Database · checked Sep 21, 2026Thanks. The Data Team will review it. | |||||
| 16 | 18 contacts → | ||||
Partial evidence “Mahzi is focused on treating under-served rare genetic neurodevelopmental disorders.” Source: RevenueBase Database · checked Sep 21, 2026Thanks. The Data Team will review it. | |||||
| 17 | 15 contacts → | ||||
Indirect evidence “Our mission is to improve the lives of those affected by Alport Syndrome through education, empowerment, advocacy, and direct investment in research.” Source: RevenueBase Database · checked Sep 21, 2026Thanks. The Data Team will review it. | |||||
| 18 | 15 contacts → | ||||
Partial evidence “The Histiocytosis Association is a global nonprofit organization dedicated to addressing the unique needs of patients and families dealing with the effects of histiocytic…” Source: RevenueBase Database · checked Sep 21, 2026Thanks. The Data Team will review it. | |||||
| 19 | 15 contacts → | ||||
Strong evidence “The Rare Cancer Research Foundation is a 501(c)3 nonprofit organization dedicated to curing rare cancers through strategic investments and innovative collaborations that…” Source: RevenueBase Database · checked Sep 21, 2026Thanks. The Data Team will review it. | |||||
| 20 | 14 contacts → | ||||
Indirect evidence “The Kelsey Research Foundation (KRF) was established on December 15, 1956, by several physicians of the Kelsey-Seybold Clinic, including John R.” Source: RevenueBase Database · checked Sep 21, 2026Thanks. The Data Team will review it. | |||||
| 21 | 14 contacts → | ||||
Strong evidence “MISSION To catalyze new treatments-and ultimately a cure-for LAM by advancing research, supporting compassionate care, and amplifying the voice of women with rare diseases.” Source: RevenueBase Database · checked Sep 21, 2026Thanks. The Data Team will review it. | |||||
| 22 | 14 contacts → | ||||
Partial evidence “Vanscoy Rare delivers hope, accelerates access and improves the outlook for people living with rare conditions.” Source: RevenueBase Database · checked Sep 21, 2026Thanks. The Data Team will review it. | |||||
| 23 | 14 contacts → | ||||
Partial evidence “Generation Patient facilitates events, online programs, and advocacy initiatives for young adults living with chronic and rare conditions to ensure that they have the…” Source: RevenueBase Database · checked Sep 21, 2026Thanks. The Data Team will review it. | |||||
| 24 | 13 contacts → | ||||
Indirect evidence “Our Mission To improve the lives of individuals with peroxisomal disorders by funding research, championing scientific collaboration, and empowering families and professionals…” Source: RevenueBase Database · checked Sep 21, 2026Thanks. The Data Team will review it. | |||||
| 25 | 11 contacts → | ||||
Partial evidence “The Desmoid Tumor Research Foundation (DTRF) is a nonprofit organization responsible for sparking the desmoid tumor research movement that has now grown into a thriving global…” Source: RevenueBase Database · checked Sep 21, 2026Thanks. The Data Team will review it. | |||||
RevenueBase Smart Search
RevenueBase Smart Search assesses information from a company’s LinkedIn profile, information supplied directly by the company, its website, and other publicly available sources. It uses that information to rate how closely the company relates to this list’s category: rare disease nonprofits. Every row carries its rating and the description the rating was read from, so each one can be checked rather than taken on trust.
- Strong evidence
- The company's own public description of its business names rare disease nonprofits.
- Partial evidence
- Its own public description names part of rare disease nonprofits, so it works in or next to the category without describing itself that way.
- Indirect evidence
- The description shown does not name rare disease nonprofits. The match rests on the rest of the company's public profile rather than the one sentence quoted here, so this is the rating to check first.
RevenueBase Smart Search is how these lists are built: RevenueBase's data engine and AI assemble the companies that match a category defined in a user's own words, so a list can be about what companies actually do rather than about whichever industry code they were filed under.
Showing 25 of 210 rare disease nonprofits based in the United States
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Rare disease nonprofits based in the United States at a glance
As of Sep 21, 2026Companies by employee count
Email recency for this list is measured worldwide rather than for United States, so it is reported there. Company counts and contact totals on this page are exact for United States, over 210 companies.
How this list was built
Company categories are rarely black and white. RevenueBase reads each company's own description of its business to decide whether it belongs among rare disease nonprofits, so some rows will be adjacent to the category rather than pure plays. The description and RevenueBase Smart Search rating on every row are there so you can check each match yourself.
- Source
- The RevenueBase company graph: 60 million companies and 398.9 million professional contacts resolved from primary sources, queried with RevenueBase Smart Search, the natural-language company search available to every RevenueBase user.
- What qualifies
- Companies headquartered in the United States whose own description of what they do matches “rare disease nonprofits”. Matching reads each company's description, not its industry code or its name.
- RevenueBase Smart Search
- RevenueBase Smart Search is how this list is assembled and how each row is rated. RevenueBase's data engine and AI assess a company's LinkedIn profile, information supplied directly by the company, its website and other publicly available information, then relate it to a category defined in a user's own words. Each row is rated strong evidence (its own description names rare disease nonprofits), partial evidence (it names part of the category) or indirect evidence (the description shown names none of it and the match rests on the rest of the company's profile). The description supporting each rating is quoted on the row.
- What we exclude
- Companies whose own description does not clearly place them among rare disease nonprofits, and records with no verified work email. Companies headquartered outside United States are excluded even when they keep an office there.
- Match quality
- In blind human judging of this matching method, 86% of matched companies were on target. Expect roughly 1 in 7 rows to be adjacent to the category rather than a pure play. Every row shows the company's own description and its RevenueBase Smart Search rating so you can judge each match yourself. Read the full methodology: how the lists are built, how the judging worked, and what the figure means.
- Ranking
- Team size, largest first: companies are ordered by the number of professionals RevenueBase tracks at each one. Employee ranges shown are reported bands, for context.
- Verification
- Every listed company carries at least one verified work email, 781 across the full list. Contacts re-verify on a rolling cadence, and each contact's own verification date is shown inside RevenueBase.
- Refresh
- This page's figures were pulled from the company graph on September 21, 2026. Lists refresh periodically as the graph is re-checked.
- Corrections
- Wrong category, wrong headquarters, or a company that has closed? Use “Report an issue” on any row. Reports go to the RevenueBase Data Team and feed back into the company graph.
Frequently asked
What is RevenueBase Smart Search?
RevenueBase Smart Search is how RevenueBase decides which companies belong on this list and how strongly each one fits. Its data engine and AI assess a company's LinkedIn profile, information supplied directly by the company, its website and other publicly available information, then relate it to the category the list is about. Each row is rated strong evidence when the company's own description names rare disease nonprofits, partial evidence when it names part of the category, and indirect evidence when the description shown names none of it and the match rests on the rest of the company's profile.
How many rare disease nonprofits are there based in the United States?
RevenueBase identified 210 rare disease nonprofits headquartered in the United States as of September 2026, matched on each company's own description of what it does rather than its industry code.
What is the largest company on this list?
Catalyst Pharmaceuticals, INC, headquartered in Coral Gables, tops this list with the largest team on record.
Which city has the most rare disease nonprofits?
Washington leads with 12 headquartered companies, followed by Austin (8) and New York (7).
How do I get contact information for these companies?
A free RevenueBase account lets you run this exact search and work the top results, verified emails and direct dials included. Upgrading unlocks all 781 verified decision-maker contacts tracked across these 210 companies.
How current is this list?
The list refreshes periodically as the company graph is re-checked. This page's figures were pulled on September 21, 2026.
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About this data
RevenueBase is a B2B data infrastructure company. Its company graph covers 60 million companies and 398.9 million professional contacts, with emails verified on a rolling cadence rather than scraped once and left to decay. These company lists are generated directly from that graph with RevenueBase Smart Search, the same natural-language company search every RevenueBase user has: describe a market in plain words, get the companies that match, and add verified emails and direct dials for the people at them. It is the same data that powers RevenueBase's B2B data products, and a free account includes 500 credits to start. How these lists are built and judged.
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