Company lists · Data refreshed September 2026
Top 25 Rare disease organizations worldwide
RevenueBase identified 783 company matches for rare disease organizations worldwide as of September 2026 and tracks 19,341 professional contacts across them, including 9,489 with verified work emails. The United States is the largest market and Paris the largest hub.
This list covers company matches for rare disease organizations worldwide, found by RevenueBase SmartSearch using the meaning of business descriptions in its company graph at Exact. Ranked by professionals tracked, largest first. Rows show recorded descriptions where available, Exact search membership and the snapshot date. Data refreshed September 21, 2026. Country and city lists for rare disease organizations follow the table.
Published by RevenueBase, a B2B data infrastructure company. This list was built with RevenueBase SmartSearch, 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 organizations worldwide
Ranked by team size| # | Company | Contacts | |||
|---|---|---|---|---|---|
| 1 | 6,136 contacts → | ||||
Exact match “Alexion, AstraZeneca Rare Disease is focused on pioneering new possibilities for the rare disease community.” Recorded in the RevenueBase company graph · Sep 21, 2026 · View company records in RevenueBase | |||||
| 2 | 1,556 contacts → | ||||
Exact match “Leading with purpose.” Recorded in the RevenueBase company graph · Sep 21, 2026 · View company records in RevenueBase | |||||
| 3 | 1,212 contacts → | ||||
Exact match “PTC is a patient-centered biopharmaceutical company focused on discovering, developing & commercializing medicine for children and adults living with rare and serious disease.” Recorded in the RevenueBase company graph · Sep 21, 2026 · View company records in RevenueBase | |||||
| 4 | 976 contacts → | ||||
Exact match “Pharming Group is a global biopharmaceutical company dedicated to transforming the lives of patients with rare, debilitating, and life-threatening diseases.” Recorded in the RevenueBase company graph · Sep 21, 2026 · View company records in RevenueBase | |||||
| 5 | 603 contacts → | ||||
Exact match “Agios is a commercial-stage biopharmaceutical company dedicated to redefining the future of rare disease treatment.” Recorded in the RevenueBase company graph · Sep 21, 2026 · View company records in RevenueBase | |||||
| 6 | 582 contacts → | ||||
Exact match “PANTHERx Rare makes rare disease care more hyper-personalized and less overwhelming by focusing relentlessly on each patient and each therapy.” Recorded in the RevenueBase company graph · Sep 21, 2026 · View company records in RevenueBase | |||||
| 7 | 571 contacts → | ||||
Exact match “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.” Recorded in the RevenueBase company graph · Sep 21, 2026 · View company records in RevenueBase | |||||
| 8 | 491 contacts → | ||||
Exact match “Our mission is to identify, develop and deliver life-changing therapies to people living with rare disease.#InRareForLife Community Guidelines…” Recorded in the RevenueBase company graph · Sep 21, 2026 · View company records in RevenueBase | |||||
| 9 | 409 contacts → | ||||
Exact match “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.” Recorded in the RevenueBase company graph · Sep 21, 2026 · View company records in RevenueBase | |||||
| 10 | 257 contacts → | ||||
Exact match “TIGEM is a leading research institute dedicated to uncovering the molecular mechanisms of rare genetic diseases and translating these discoveries into innovative therapies, with a…” Recorded in the RevenueBase company graph · Sep 21, 2026 · View company records in RevenueBase | |||||
| 11 | 217 contacts → | ||||
Exact match “Focused on complex therapies and orphan diseases, Varifarma is a company which is devoted to improving the patients' quality of life.” Recorded in the RevenueBase company graph · Sep 21, 2026 · View company records in RevenueBase | |||||
| 12 | 196 contacts → | ||||
Exact match “We’re committed to supporting patients and their caregivers in every way we can.” Recorded in the RevenueBase company graph · Sep 21, 2026 · View company records in RevenueBase | |||||
| 13 | 185 contacts → | ||||
Exact match Recorded description not available. Recorded in the RevenueBase company graph · Sep 21, 2026 · View company records in RevenueBase | |||||
| 14 | 168 contacts → | ||||
Exact match “Bionews is a digital health solutions company dedicated to empowering more than 50 rare disease communities with trusted information, news, and connections.” Recorded in the RevenueBase company graph · Sep 21, 2026 · View company records in RevenueBase | |||||
| 15 | 156 contacts → | ||||
Exact match “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…” Recorded in the RevenueBase company graph · Sep 21, 2026 · View company records in RevenueBase | |||||
| 16 | 118 contacts → | ||||
Exact match “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…” Recorded in the RevenueBase company graph · Sep 21, 2026 · View company records in RevenueBase | |||||
| 17 | 110 contacts → | ||||
Exact match “Ågrenska is a national centre of competence for rare diseases and other disabilities.” Recorded in the RevenueBase company graph · Sep 21, 2026 · View company records in RevenueBase | |||||
| 18 | 84 contacts → | ||||
Exact match “We are a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined diseases in areas of high unmet medical need, with an initial…” Recorded in the RevenueBase company graph · Sep 21, 2026 · View company records in RevenueBase | |||||
| 19 | 83 contacts → | ||||
Exact match “Amryt Pharma was acquired by Chiesi Farmaceutici S.p.A.(“Chiesi”) April 12, 2023.” Recorded in the RevenueBase company graph · Sep 21, 2026 · View company records in RevenueBase | |||||
| 20 | 81 contacts → | ||||
Exact match “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…” Recorded in the RevenueBase company graph · Sep 21, 2026 · View company records in RevenueBase | |||||
| 21 | 78 contacts → | ||||
Exact match “We are a purpose-driven, commercial-stage company with a late-stage pipeline committed to redefining what is possible in bringing life-changing therapies to people living with…” Recorded in the RevenueBase company graph · Sep 21, 2026 · View company records in RevenueBase | |||||
| 22 | 75 contacts → | ||||
Exact match “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…” Recorded in the RevenueBase company graph · Sep 21, 2026 · View company records in RevenueBase | |||||
| 23 | 70 contacts → | ||||
Exact match “We are committed to transforming the lives of people affected by cancer or rare disease, globally.” Recorded in the RevenueBase company graph · Sep 21, 2026 · View company records in RevenueBase | |||||
| 24 | 65 contacts → | ||||
Exact match “ITF Therapeutics is the US-based rare disease division of Italfarmaco S.p.A.” Recorded in the RevenueBase company graph · Sep 21, 2026 · View company records in RevenueBase | |||||
| 25 | 63 contacts → | ||||
Exact match “This is a time of great hope for individuals with complex diseases such as NF1 and their families.” Recorded in the RevenueBase company graph · Sep 21, 2026 · View company records in RevenueBase | |||||
RevenueBase SmartSearch
Describe a niche business in your own words. RevenueBase SmartSearch compares the meaning of your description with company business descriptions compiled in the RevenueBase company graph. Exact, Similar, Related and Broad control how closely companies must fit.
This list uses Exact for “rare disease organizations”. Each Exact match badge shows membership in that saved search; it is not an individual similarity score or a service-quality rating. Recorded descriptions are shown where available.
Showing 25 of 783 rare disease organizations worldwide
See the full list, filter by size, metro and specialty, and access 9,489 tracked professional contacts with verified work emails. Direct dials are available where recorded.
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Rare disease organizations worldwide at a glance
As of Sep 21, 2026Companies by employee count
Email recency is reported on each country list linked below.
How this list was built
The recorded descriptions help you assess these company matches. Check a company’s website for its current services and locations.
- Source
- The RevenueBase company graph: 60 million companies and 398.9 million professional contacts resolved from primary sources, queried with RevenueBase SmartSearch, the natural-language company search available to every RevenueBase user.
- Coverage
- Every country. Country and city lists for rare disease organizations are linked below.
- What qualifies
- Company records worldwide whose business descriptions match “rare disease organizations” by meaning at the Exact setting. Descriptions are compiled in the RevenueBase company graph from public websites, profiles and company information where available; they are not guaranteed quotations from the company's website.
- RevenueBase SmartSearch
- Describe a niche company in your own words to find businesses by meaning. Exact, Similar, Related and Broad control the breadth of RevenueBase SmartSearch. This saved list uses Exact. Each row's Exact match badge shows inclusion in that search, not an individual score or a service-quality judgment.
- Public preview
- The public preview omits records without a verified work email. Dataset totals and geographic summaries describe the full matched records before these preview filters.
- Match quality
- RevenueBase's August 25, 2026 study assessed 4,198 description/company pairs across matching settings. The publisher reports 86% good or better at Exact. This evaluates the method across queries; it does not audit this individual list. Recorded company descriptions, where available, help you judge each match yourself. Read the full methodology: how the lists are built and what the study measures.
- 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 displayed company carries at least one verified work email. RevenueBase tracks 9,489 professional contacts with verified work emails across the full matched dataset. 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.
- Record context
- Company records and descriptions can change. Check the company's website for its current services and locations.
Rare disease organizations by location
4 lists · largest firstEvery Rare disease organizations 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 SmartSearch?
RevenueBase SmartSearch finds niche companies by the meaning of their business descriptions in the RevenueBase company graph. Exact, Similar, Related and Broad control how closely companies must fit your description. This list uses Exact for “rare disease organizations”; each Exact match badge shows search membership, not an individual similarity or quality score.
How many rare disease organizations are there worldwide?
RevenueBase identified 783 company matches for rare disease organizations worldwide as of September 2026, using the meaning of business descriptions at Exact. This is the recorded matched set in the RevenueBase company graph.
What is the largest company on this list?
Alexion Pharmaceuticals, INC tops the displayed preview by professional contacts tracked in RevenueBase. Recorded headquarters appear in the company rows.
Which country has the most rare disease organizations?
The United States leads with 223 companies, followed by France (72) and the United Kingdom (47). Every rare disease organizations list by country and city is linked on this page.
Which city has the most rare disease organizations?
Paris leads with 21 company records, followed by Cambridge (12) and London (12).
How do I get contact information for these companies?
A free RevenueBase account lets you run this exact search and work limited free results with 500 credits to start. Upgrading provides access to the 9,489 professional contacts with verified work emails tracked across these 783 companies; direct dials are available where recorded.
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.
Ready to reach these companies? Verified emails and direct dials for all 783 rare disease organizations worldwide. Start with a free account, no credit card.
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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 SmartSearch, 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.
More company lists
Company intelligence · public preview
Top 25 rare disease organizations worldwide
Find companies in any niche.
Describe the businesses you want in your own words. RevenueBase SmartSearch matches company descriptions by meaning.
RevenueBase records 783 matching company records for rare disease organizations worldwide, with 19,341 tracked contacts and 9,489 verified work emails.
This public preview shows 25 selected records ordered by tracked contacts. RevenueBase SmartSearch finds company descriptions that match by meaning at Exact. Snapshot: September 21, 2026.
RevenueBase compiles business descriptions for niche-company discovery; each row shows a recorded excerpt where available. Adapt this search to find the companies and contacts you want.
Top 25 rare disease organizations worldwide
The public list shows selected company records with contact and verified-email coverage. Reported employee bands provide separate company-size context.
| # | Company | Headquarters on record | Reported employees | RevenueBase SmartSearch | Tracked contacts |
|---|---|---|---|---|---|
| 1 |  Alexion Pharmaceuticals, INCalexion.comHQ: Boston, MA, United StatesReported employees: 1,001-5,000 | Boston, MA, United States | 1,001-5,000 | Exact match | 6,136 contacts → |
“Alexion, AstraZeneca Rare Disease is focused on pioneering new possibilities for the rare disease community.” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 2 | Ultragenyxultragenyx.com HQ: Novato, CA, United StatesReported employees: 1,001-5,000 | Novato, CA, United States | 1,001-5,000 | Exact match | 1,556 contacts → |
“Leading with purpose.” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 3 |  PTC Therapeutics, INCptcbio.comHQ: Warren, NJ, United StatesReported employees: 501-1,000 | Warren, NJ, United States | 501-1,000 | Exact match | 1,212 contacts → |
“PTC is a patient-centered biopharmaceutical company focused on discovering, developing & commercializing medicine for children and adults living with rare and serious disease.” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 4 | Pharming Grouppharming.com HQ: Leiden, NetherlandsReported employees: 201-500 | Leiden, Netherlands | 201-500 | Exact match | 976 contacts → |
“Pharming Group is a global biopharmaceutical company dedicated to transforming the lives of patients with rare, debilitating, and life-threatening diseases.” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 5 |  Agios Pharmaceuticalsagios.comHQ: Cambridge, MA, United StatesReported employees: 501-1,000 | Cambridge, MA, United States | 501-1,000 | Exact match | 603 contacts → |
“Agios is a commercial-stage biopharmaceutical company dedicated to redefining the future of rare disease treatment.” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 6 |  Pantherx Rare Pharmacypantherxrare.comHQ: Pittsburgh, PA, United StatesReported employees: 501-1,000 | Pittsburgh, PA, United States | 501-1,000 | Exact match | 582 contacts → |
“PANTHERx Rare makes rare disease care more hyper-personalized and less overwhelming by focusing relentlessly on each patient and each therapy.” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 7 |  Afm-Téléthonafm-telethon.frHQ: Evry, FranceReported employees: 201-500 | Evry, France | 201-500 | Exact match | 571 contacts → |
“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.” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 8 | Travere Therapeuticstravere.com HQ: San Diego, CA, United StatesReported employees: 201-500 | San Diego, CA, United States | 201-500 | Exact match | 491 contacts → |
“Our mission is to identify, develop and deliver life-changing therapies to people living with rare disease.#InRareForLife Community Guidelines…” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 9 | Lifearclifearc.org HQ: London, United KingdomReported employees: 201-500 | London, United Kingdom | 201-500 | Exact match | 409 contacts → |
“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.” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 10 | HQ: Pozzuoli, ItalyReported employees: 201-500 | Pozzuoli, Italy | 201-500 | Exact match | 257 contacts → |
“TIGEM is a leading research institute dedicated to uncovering the molecular mechanisms of rare genetic diseases and translating these discoveries into innovative therapies, with a…” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 11 | Laboratorio Varifarmavarifarma.com HQ: Beccar, ArgentinaReported employees: 201-500 | Beccar, Argentina | 201-500 | Exact match | 217 contacts → |
“Focused on complex therapies and orphan diseases, Varifarma is a company which is devoted to improving the patients' quality of life.” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 12 | Cycle Pharmaceuticalscyclepharma.com HQ: Cambridge, United KingdomReported employees: 51-200 | Cambridge, United Kingdom | 51-200 | Exact match | 196 contacts → |
“We’re committed to supporting patients and their caregivers in every way we can.” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 13 | Catalyst Pharmaceuticals, INCcatalystpharma.com HQ: Coral Gables, FL, United StatesReported employees: 51-200 | Coral Gables, FL, United States | 51-200 | Exact match | 185 contacts → |
No recorded description excerpt is available for this public record. | |||||
| 14 | Bionews, INCbionews.com HQ: Pensacola, FL, United StatesReported employees: 51-200 | Pensacola, FL, United States | 51-200 | Exact match | 168 contacts → |
“Bionews is a digital health solutions company dedicated to empowering more than 50 rare disease communities with trusted information, news, and connections.” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 15 | National Organization For Rare Disordersrarediseases.org HQ: Danbury, CT, United StatesReported employees: 51-200 | Danbury, CT, United States | 51-200 | Exact match | 156 contacts → |
“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…” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 16 | Orphalanorphalan.com HQ: Paris, FranceReported employees: 51-200 | Paris, France | 51-200 | Exact match | 118 contacts → |
“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…” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 17 |  Ågrenskaagrenska.seHQ: Hovås, SwedenReported employees: 201-500 | Hovås, Sweden | 201-500 | Exact match | 110 contacts → |
“Ågrenska is a national centre of competence for rare diseases and other disabilities.” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 18 | Fulcrum Therapeuticsfulcrumtx.com HQ: Cambridge, MA, United StatesReported employees: 51-200 | Cambridge, MA, United States | 51-200 | Exact match | 84 contacts → |
“We are a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined diseases in areas of high unmet medical need, with an initial…” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 19 | Amryt Pharmachiesirarediseases.com HQ: Dublin City, United StatesReported employees: 201-500 | Dublin City, United States | 201-500 | Exact match | 83 contacts → |
“Amryt Pharma was acquired by Chiesi Farmaceutici S.p.A.(“Chiesi”) April 12, 2023.” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 20 | Everylife Foundation For Rare Diseaseseverylifefoundation.org HQ: Washington, DC, United StatesReported employees: 11-50 | Washington, DC, United States | 11-50 | Exact match | 81 contacts → |
“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…” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 21 |  Zevra Therapeuticszevra.comHQ: Boston, MA, United StatesReported employees: 51-200 | Boston, MA, United States | 51-200 | Exact match | 78 contacts → |
“We are a purpose-driven, commercial-stage company with a late-stage pipeline committed to redefining what is possible in bringing life-changing therapies to people living with…” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 22 | Eurordis-Rare Diseases Europeeurordis.org HQ: Paris, FranceReported employees: 11-50 | Paris, France | 11-50 | Exact match | 75 contacts → |
“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…” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 23 | Eusa Pharmaeusapharma.com HQ: Hemel Hempstead, United KingdomReported employees: 201-500 | Hemel Hempstead, United Kingdom | 201-500 | Exact match | 70 contacts → |
“We are committed to transforming the lives of people affected by cancer or rare disease, globally.” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 24 | ITF Therapeutics LLCitftherapeutics.com HQ: Concord, MA, United StatesReported employees: 51-200 | Concord, MA, United States | 51-200 | Exact match | 65 contacts → |
“ITF Therapeutics is the US-based rare disease division of Italfarmaco S.p.A.” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 25 | Ihealth And Wellness Foundation, INCihealthwellness.org HQ: New York, NY, United StatesReported employees: 11-50 | New York, NY, United States | 11-50 | Exact match | 63 contacts → |
“This is a time of great hope for individuals with complex diseases such as NF1 and their families.” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
Showing 25 of 783 company matches
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Rare disease organizations: dataset insights
September 21, 2026These summaries describe recorded coverage in this saved dataset. This page is derived from saved source records; per-contact mobile coverage and email-recency results are not established by this snapshot.
Reported company sizes
782 records in the displayed employee bands.
1 matching records are outside the displayed bands or do not have a displayed size. Bars show shares of the displayed bands.
Email recency for this list is measured across the whole country list rather than for Worldwide, so it is reported there. Company counts and contact totals on this page are exact for Worldwide, over 783 companies.
How this company list was built
Describe a niche in your own words: companies installing commercial solar, or manufacturers of sustainable packaging. RevenueBase SmartSearch finds companies whose compiled business descriptions match by meaning. This list is one saved example to adapt.
Where the company descriptions come from
RevenueBase compiles company information from public sources, including company websites and public professional profiles such as LinkedIn, alongside information supplied by companies. The short recorded excerpt shown here is separate from the complete business description used for matching; individual source URLs and collection dates are not provided in this public snapshot.
RevenueBase SmartSearch matching settings
- Exact
- Closest fits to the business described.
- Similar
- Similar businesses with a wider range of fits.
- Related
- Related businesses in and around the category.
- Broad
- A broader set of businesses connected to the description.
RevenueBase SmartSearch · Exact match identifies inclusion in this search's closest semantic matching setting. It is not a per-company confidence score, a literal keyword match, or a quality endorsement.
Selection, ordering and scope
The public list shows selected company records with contact and verified-email coverage. Reported employee bands provide separate company-size context.
This page is derived from saved source records; per-contact mobile coverage and email-recency results are not established by this snapshot.
Recorded company descriptions support research; check company websites for current services. The selected preview is ordered by tracked contacts and is not a list of the best service providers.
Snapshot: September 21, 2026.
RevenueBase is a B2B data infrastructure company. Its September 3, 2026 published methodology describes a graph of 60 million companies and 398.9 million professional contacts; these platform figures differ from this page's totals. Published methodology ↗
Rare disease organizations by location
4 lists · largest firstEvery Rare disease organizations list RevenueBase publishes, by country and city. Counts are matching companies in each list as of its own refresh date.
Countries
Questions about this company list
What does Exact match mean for rare disease organizations?
The separate RevenueBase SmartSearch column identifies inclusion at the closest semantic matching preset. It is not a literal keyword match, a per-company confidence score or a quality endorsement. Changing the search form opens a new search in RevenueBase; it does not reclassify these saved rows.
How many rare disease organizations did RevenueBase find worldwide?
The saved snapshot records 783 matching company records, 19,341 tracked contacts and 9,489 verified emails. The public preview displays 25 selected records.
Where do the company descriptions come from?
RevenueBase compiles company information from public sources, including company websites and public professional profiles such as LinkedIn, alongside information supplied by companies. The short recorded excerpt shown here is separate from the complete business description used for matching; individual source URLs and collection dates are not provided in this public snapshot.
How are the public companies ordered?
The selected public records are ordered by contacts tracked in RevenueBase, highest first. Contact coverage is a prospecting measure, not an assessment of the company's services or quality.
How can I get contact information?
Select a company's contact count to open its filtered contacts in RevenueBase. Free signup is required and includes 500 credits and limited free results. Verified emails, direct dials and mobile numbers depend on available coverage.
How current is this list?
Snapshot: September 21, 2026. Review each company's website for current services and locations. A recorded business description does not establish that every company offers every service named in the search.
What do headquarters and employee bands mean?
These are recorded company-level fields, rather than a map of every branch or a count of specialists. Missing headquarters or employee bands are labeled; older records may show a location separately from recorded headquarters.
Build your next target company list
Adapt this niche search and work with available contacts in RevenueBase.
Related lists
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Cite this list
RevenueBase Data Team. Top 25 rare disease organizations worldwide. Snapshot: September 21, 2026. https://revenuebase.ai/companies/browse/rare-disease-organizations.
This is a selected public preview of saved company records, rather than an independently verified census. Open the source page ↗
More company lists
Company intelligence · public preview
Top 25 rare disease organizations worldwide
RevenueBase records 783 matching company records for rare disease organizations worldwide, with 19,341 tracked contacts and 9,489 verified work emails.
This public preview shows 25 selected records ordered by tracked contacts. RevenueBase SmartSearch finds company descriptions that match by meaning at Exact. Snapshot: September 21, 2026.
RevenueBase compiles business descriptions for niche-company discovery; each row shows a recorded excerpt where available. Adapt this search to find the companies and contacts you want.
Top 25 rare disease organizations worldwide
The public list shows selected company records with contact and verified-email coverage. Reported employee bands provide separate company-size context.
| # | Company | Headquarters on record | Reported employees | RevenueBase SmartSearch | Tracked contacts |
|---|---|---|---|---|---|
| 1 | Alexion Pharmaceuticals, INCalexion.com HQ: Boston, MA, United StatesReported employees: 1,001-5,000 | Boston, MA, United States | 1,001-5,000 | Exact match | 6,136 contacts → |
“Alexion, AstraZeneca Rare Disease is focused on pioneering new possibilities for the rare disease community.” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 2 | Ultragenyxultragenyx.com HQ: Novato, CA, United StatesReported employees: 1,001-5,000 | Novato, CA, United States | 1,001-5,000 | Exact match | 1,556 contacts → |
“Leading with purpose.” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 3 | PTC Therapeutics, INCptcbio.com HQ: Warren, NJ, United StatesReported employees: 501-1,000 | Warren, NJ, United States | 501-1,000 | Exact match | 1,212 contacts → |
“PTC is a patient-centered biopharmaceutical company focused on discovering, developing & commercializing medicine for children and adults living with rare and serious disease.” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 4 | Pharming Grouppharming.com HQ: Leiden, NetherlandsReported employees: 201-500 | Leiden, Netherlands | 201-500 | Exact match | 976 contacts → |
“Pharming Group is a global biopharmaceutical company dedicated to transforming the lives of patients with rare, debilitating, and life-threatening diseases.” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 5 | Agios Pharmaceuticalsagios.com HQ: Cambridge, MA, United StatesReported employees: 501-1,000 | Cambridge, MA, United States | 501-1,000 | Exact match | 603 contacts → |
“Agios is a commercial-stage biopharmaceutical company dedicated to redefining the future of rare disease treatment.” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 6 | Pantherx Rare Pharmacypantherxrare.com HQ: Pittsburgh, PA, United StatesReported employees: 501-1,000 | Pittsburgh, PA, United States | 501-1,000 | Exact match | 582 contacts → |
“PANTHERx Rare makes rare disease care more hyper-personalized and less overwhelming by focusing relentlessly on each patient and each therapy.” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 7 | Afm-Téléthonafm-telethon.fr HQ: Evry, FranceReported employees: 201-500 | Evry, France | 201-500 | Exact match | 571 contacts → |
“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.” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 8 | Travere Therapeuticstravere.com HQ: San Diego, CA, United StatesReported employees: 201-500 | San Diego, CA, United States | 201-500 | Exact match | 491 contacts → |
“Our mission is to identify, develop and deliver life-changing therapies to people living with rare disease.#InRareForLife Community Guidelines…” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 9 | Lifearclifearc.org HQ: London, United KingdomReported employees: 201-500 | London, United Kingdom | 201-500 | Exact match | 409 contacts → |
“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.” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 10 | HQ: Pozzuoli, ItalyReported employees: 201-500 | Pozzuoli, Italy | 201-500 | Exact match | 257 contacts → |
“TIGEM is a leading research institute dedicated to uncovering the molecular mechanisms of rare genetic diseases and translating these discoveries into innovative therapies, with a…” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 11 | Laboratorio Varifarmavarifarma.com HQ: Beccar, ArgentinaReported employees: 201-500 | Beccar, Argentina | 201-500 | Exact match | 217 contacts → |
“Focused on complex therapies and orphan diseases, Varifarma is a company which is devoted to improving the patients' quality of life.” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 12 | Cycle Pharmaceuticalscyclepharma.com HQ: Cambridge, United KingdomReported employees: 51-200 | Cambridge, United Kingdom | 51-200 | Exact match | 196 contacts → |
“We’re committed to supporting patients and their caregivers in every way we can.” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 13 | Catalyst Pharmaceuticals, INCcatalystpharma.com HQ: Coral Gables, FL, United StatesReported employees: 51-200 | Coral Gables, FL, United States | 51-200 | Exact match | 185 contacts → |
No recorded description excerpt is available for this public record. | |||||
| 14 | Bionews, INCbionews.com HQ: Pensacola, FL, United StatesReported employees: 51-200 | Pensacola, FL, United States | 51-200 | Exact match | 168 contacts → |
“Bionews is a digital health solutions company dedicated to empowering more than 50 rare disease communities with trusted information, news, and connections.” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 15 | National Organization For Rare Disordersrarediseases.org HQ: Danbury, CT, United StatesReported employees: 51-200 | Danbury, CT, United States | 51-200 | Exact match | 156 contacts → |
“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…” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 16 | Orphalanorphalan.com HQ: Paris, FranceReported employees: 51-200 | Paris, France | 51-200 | Exact match | 118 contacts → |
“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…” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 17 | Ågrenskaagrenska.se HQ: Hovås, SwedenReported employees: 201-500 | Hovås, Sweden | 201-500 | Exact match | 110 contacts → |
“Ågrenska is a national centre of competence for rare diseases and other disabilities.” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 18 | Fulcrum Therapeuticsfulcrumtx.com HQ: Cambridge, MA, United StatesReported employees: 51-200 | Cambridge, MA, United States | 51-200 | Exact match | 84 contacts → |
“We are a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined diseases in areas of high unmet medical need, with an initial…” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 19 | Amryt Pharmachiesirarediseases.com HQ: Dublin City, United StatesReported employees: 201-500 | Dublin City, United States | 201-500 | Exact match | 83 contacts → |
“Amryt Pharma was acquired by Chiesi Farmaceutici S.p.A.(“Chiesi”) April 12, 2023.” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 20 | Everylife Foundation For Rare Diseaseseverylifefoundation.org HQ: Washington, DC, United StatesReported employees: 11-50 | Washington, DC, United States | 11-50 | Exact match | 81 contacts → |
“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…” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 21 | Zevra Therapeuticszevra.com HQ: Boston, MA, United StatesReported employees: 51-200 | Boston, MA, United States | 51-200 | Exact match | 78 contacts → |
“We are a purpose-driven, commercial-stage company with a late-stage pipeline committed to redefining what is possible in bringing life-changing therapies to people living with…” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 22 | Eurordis-Rare Diseases Europeeurordis.org HQ: Paris, FranceReported employees: 11-50 | Paris, France | 11-50 | Exact match | 75 contacts → |
“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…” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 23 | Eusa Pharmaeusapharma.com HQ: Hemel Hempstead, United KingdomReported employees: 201-500 | Hemel Hempstead, United Kingdom | 201-500 | Exact match | 70 contacts → |
“We are committed to transforming the lives of people affected by cancer or rare disease, globally.” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 24 | ITF Therapeutics LLCitftherapeutics.com HQ: Concord, MA, United StatesReported employees: 51-200 | Concord, MA, United States | 51-200 | Exact match | 65 contacts → |
“ITF Therapeutics is the US-based rare disease division of Italfarmaco S.p.A.” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 25 | Ihealth And Wellness Foundation, INCihealthwellness.org HQ: New York, NY, United StatesReported employees: 11-50 | New York, NY, United States | 11-50 | Exact match | 63 contacts → |
“This is a time of great hope for individuals with complex diseases such as NF1 and their families.” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
Showing 25 of 783 company matches
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Rare disease organizations: dataset insights
September 21, 2026These summaries describe recorded coverage in this saved dataset. This page is derived from saved source records; per-contact mobile coverage and email-recency results are not established by this snapshot.
Reported company sizes
782 records in the displayed employee bands.
1 matching records are outside the displayed bands or do not have a displayed size. Bars show shares of the displayed bands.
Email recency for this list is measured across the whole country list rather than for Worldwide, so it is reported there. Company counts and contact totals on this page are exact for Worldwide, over 783 companies.
How this company list was built
Describe a niche in your own words: companies installing commercial solar, or manufacturers of sustainable packaging. RevenueBase SmartSearch finds companies whose compiled business descriptions match by meaning. This list is one saved example to adapt.
Where the company descriptions come from
RevenueBase compiles company information from public sources, including company websites and public professional profiles such as LinkedIn, alongside information supplied by companies. The short recorded excerpt shown here is separate from the complete business description used for matching; individual source URLs and collection dates are not provided in this public snapshot.
RevenueBase SmartSearch matching settings
- Exact
- Closest fits to the business described.
- Similar
- Similar businesses with a wider range of fits.
- Related
- Related businesses in and around the category.
- Broad
- A broader set of businesses connected to the description.
RevenueBase SmartSearch · Exact match identifies inclusion in this search's closest semantic matching setting. It is not a per-company confidence score, a literal keyword match, or a quality endorsement.
Selection, ordering and scope
The public list shows selected company records with contact and verified-email coverage. Reported employee bands provide separate company-size context.
This page is derived from saved source records; per-contact mobile coverage and email-recency results are not established by this snapshot.
Recorded company descriptions support research; check company websites for current services. The selected preview is ordered by tracked contacts and is not a list of the best service providers.
Snapshot: September 21, 2026.
RevenueBase is a B2B data infrastructure company. Its September 3, 2026 published methodology describes a graph of 60 million companies and 398.9 million professional contacts; these platform figures differ from this page's totals. Published methodology ↗
Rare disease organizations by location
4 lists · largest firstEvery Rare disease organizations list RevenueBase publishes, by country and city. Counts are matching companies in each list as of its own refresh date.
Countries
Questions about this company list
What does Exact match mean for rare disease organizations?
The separate RevenueBase SmartSearch column identifies inclusion at the closest semantic matching preset. It is not a literal keyword match, a per-company confidence score or a quality endorsement. Changing the search form opens a new search in RevenueBase; it does not reclassify these saved rows.
How many rare disease organizations did RevenueBase find worldwide?
The saved snapshot records 783 matching company records, 19,341 tracked contacts and 9,489 verified emails. The public preview displays 25 selected records.
Where do the company descriptions come from?
RevenueBase compiles company information from public sources, including company websites and public professional profiles such as LinkedIn, alongside information supplied by companies. The short recorded excerpt shown here is separate from the complete business description used for matching; individual source URLs and collection dates are not provided in this public snapshot.
How are the public companies ordered?
The selected public records are ordered by contacts tracked in RevenueBase, highest first. Contact coverage is a prospecting measure, not an assessment of the company's services or quality.
How can I get contact information?
Select a company's contact count to open its filtered contacts in RevenueBase. Free signup is required and includes 500 credits and limited free results. Verified emails, direct dials and mobile numbers depend on available coverage.
How current is this list?
Snapshot: September 21, 2026. Review each company's website for current services and locations. A recorded business description does not establish that every company offers every service named in the search.
What do headquarters and employee bands mean?
These are recorded company-level fields, rather than a map of every branch or a count of specialists. Missing headquarters or employee bands are labeled; older records may show a location separately from recorded headquarters.
Build your next target company list
Adapt this niche search and work with available contacts in RevenueBase.
Related lists
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Cite this list
RevenueBase Data Team. Top 25 rare disease organizations worldwide. Snapshot: September 21, 2026. https://revenuebase.ai/companies/browse/rare-disease-organizations.
This is a selected public preview of saved company records, rather than an independently verified census. Open the source page ↗