Company lists · Data refreshed September 2026

Top 25 Rare disease nonprofits worldwide

RevenueBase identified 672 rare disease nonprofits worldwide as of September 2026 and tracks 5,955 professional contacts across them, including 1,935 with verified work emails. The United States is the largest market and Paris the largest hub.

This list covers rare disease nonprofits worldwide, 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. Country and city lists for rare disease nonprofits follow the table.

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.

How to read this list HQ checked on every row RevenueBase Smart Search rating on every row Refreshed Sep 21, 2026 86% judged on-target in blind review
672Companies
5,955Contacts tracked
1,935Verified emails
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The 25 largest rare disease nonprofits worldwide

Ranked by team size
Top 25 of 672 rare disease nonprofits worldwide, ranked by team size, largest first. Data refreshed Sep 21, 2026. RevenueBase Smart Search rates how each company's information relates to rare disease nonprofits: strong evidence (its own description names it), partial (names part of it), indirect (matched on the rest of its profile rather than the description shown). Click a contact count to view that company's people.
#CompanyRevenueBase Smart SearchHeadquartersEmployeesContacts
1Partial evidenceEvry, FranceHQ checked Sep 21, 2026201-500571 contacts →

Partial evidence

“L’AFM-Téléthon est une association de parents et de malades qui mène un combat sans relâche contre des maladies génétiques, rares et lourdement invalidantes.”

Source: RevenueBase Database · checked Sep 21, 2026
2Strong evidenceLondon, United KingdomHQ checked Sep 21, 2026201-500409 contacts →

Strong evidence

“LifeArc® is a self-funded medical research organisation with one clear purpose – transforming the lives of people living with rare diseases and drug-resistant infections.”

Source: RevenueBase Database · checked Sep 21, 2026
3Indirect evidenceCoral Gables, FL, United StatesHQ checked Sep 21, 202651-200185 contacts →

Indirect evidence

Source: RevenueBase Database · checked Sep 21, 2026
4Strong evidencePensacola, FL, United StatesHQ checked Sep 21, 202651-200168 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, 2026
5Strong evidenceDanbury, CT, United StatesHQ checked Sep 21, 202651-200156 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, 2026
6Strong evidenceParis, FranceHQ checked Sep 21, 202651-200118 contacts →

Strong evidence

“Transforming Rare Disease Treatment At Orphalan we identify, develop, and provide access to innovative treatments for patients with rare diseases to make a positive and meaningful…”

Source: RevenueBase Database · checked Sep 21, 2026
7Strong evidenceHovås, SwedenHQ checked Sep 21, 2026201-500110 contacts →

Strong evidence

“Ågrenska is a national centre of competence for rare diseases and other disabilities.”

Source: RevenueBase Database · checked Sep 21, 2026
8Strong evidenceWashington, DC, United StatesHQ checked Sep 21, 202611-5081 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, 2026
9Strong evidenceParis, FranceHQ checked Sep 21, 202611-5075 contacts →

Strong evidence

“EURORDIS-Rare Diseases Europe is a unique, non-profit alliance of over rare disease patient organisations from more than 70 countries that work together to improve the lives of…”

Source: RevenueBase Database · checked Sep 21, 2026
10Indirect evidenceNovato, CA, United StatesHQ checked Sep 21, 20261,001-5,00067 contacts →

Indirect evidence

“Leading with purpose.”

Source: RevenueBase Database · checked Sep 21, 2026
11Partial evidenceNew York, NY, United StatesHQ checked Sep 21, 202611-5063 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, 2026
12Indirect evidenceMadrid, SpainHQ checked Sep 21, 202651-20053 contacts →

Indirect evidence

“Sobi es una compañía biofarmacéutica internacional que trabaja cada día para ofrecer terapias y servicios innovadores que mejoren la vida de las personas con enfermedades raras y…”

Source: RevenueBase Database · checked Sep 21, 2026
13Indirect evidenceMadrid, SpainHQ checked Sep 21, 202651-20049 contacts →

Indirect evidence

“La Federación Española de Enfermedades Raras (FEDER) nació en con el objetivo de ser la esperanza de más de tres millones de personas que conviven con estas patologías en nuestro…”

Source: RevenueBase Database · checked Sep 21, 2026
14Strong evidencePhiladelphia, PA, United StatesHQ checked Sep 21, 2026n/a48 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, 2026
15Indirect evidenceMadrid, SpainHQ checked Sep 21, 202651-20048 contacts →

Indirect evidence

“The CIBERER is a cooperative network structure, with 60 research groups located in the main research centres all over Spain.”

Source: RevenueBase Database · checked Sep 21, 2026
16Strong evidenceMidlothian, VA, United StatesHQ checked Sep 21, 202611-5046 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, 2026
17Partial evidenceSydney, AustraliaHQ checked Sep 21, 202611-5042 contacts →

Partial evidence

Rare Cancers Australia (RCA) is a leading for-impact charity that works to improve the lives and health outcomes of Australian patients, carers and families affected by a rare or…”

Source: RevenueBase Database · checked Sep 21, 2026
18Indirect evidenceLisbon, PortugalHQ checked Sep 21, 202651-20041 contacts →

Indirect evidence

“A Raríssimas – Associação Nacional de Deficiências Mentais e Raras é uma IPSS, fundada em Abril de 2002, que desenvolve a sua acção junto de doentes e familiares que convivem de…”

Source: RevenueBase Database · checked Sep 21, 2026
19Strong evidenceWashington, United StatesHQ checked Sep 21, 202611-5039 contacts →

Strong evidence

“Empowering the Next Generation Rare Disease Advocate.”

Source: RevenueBase Database · checked Sep 21, 2026
20Partial evidenceLaxou, FranceHQ checked Sep 21, 202611-5038 contacts →

Partial evidence

“Fondée en 1992 par Guy Alba, l’Association Européenne contre les Leucodystrophies (ELA) regroupe des familles qui se mobilisent pour vaincre ces maladies génétiques rares qui…”

Source: RevenueBase Database · checked Sep 21, 2026
21Indirect evidenceCiudad De Buenos Aires, ArgentinaHQ checked Sep 21, 202611-5038 contacts →

Indirect evidence

“Fundación SPINE es una asociación sin fines de lucro que realiza investigación y tratamiento de pacientes con enfermedades raras o poco frecuentes, con el objetivo de mejorar su…”

Source: RevenueBase Database · checked Sep 21, 2026
22Strong evidenceLong Beach, CA, United StatesHQ checked Sep 21, 202651-20036 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, 2026
23Strong evidenceDowney, CA, United StatesHQ checked Sep 21, 202651-20031 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, 2026
24Partial evidenceWashington, DC, United StatesHQ checked Sep 21, 202611-5028 contacts →

Partial evidence

“New advanced medicines are curing patients of serious diseases.”

Source: RevenueBase Database · checked Sep 21, 2026
25Strong evidenceLondon, United KingdomHQ checked Sep 21, 202611-5028 contacts →

Strong evidence

“At Partners4Access, we believe patients with rare diseases deserve the right to access the most appropriate treatment.”

Source: RevenueBase Database · checked Sep 21, 2026

Showing 25 of 672 rare disease nonprofits worldwide

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Rare disease nonprofits worldwide at a glance

As of Sep 21, 2026
32.5%Verified-email coverage
8.9Average contacts per company
1-10Median size (employees)
2.4%Largest-city concentration · Paris
Sep 21, 2026Page data refreshed

Companies by employee count

1-10
500
11-50
125
51-200
31
201-500
7
501-1,000
3
1,001-5,000
4
5,001-10,000
1
10001+
1

Email recency is reported on each country list linked below.

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.
Coverage
Every country. Country and city lists for rare disease nonprofits are linked below.
What qualifies
Companies worldwide 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.
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, 1,935 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.

Rare disease nonprofits by location

3 lists · largest first

Every Rare disease nonprofits list RevenueBase publishes, by country and city. Counts are matching companies in each list as of its own refresh date.

Countries

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 worldwide?

RevenueBase identified 672 rare disease nonprofits worldwide 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?

Afm-Téléthon, headquartered in Cedex, tops this list with the largest team on record.

Which country has the most rare disease nonprofits?

The United States leads with 210 companies, followed by France (46) and the United Kingdom (34). Every rare disease nonprofits list by country and city is linked on this page.

Which city has the most rare disease nonprofits?

Paris leads with 16 headquartered companies, followed by Washington (12) and Madrid (9).

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 1,935 verified decision-maker contacts tracked across these 672 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.