Company lists · Data refreshed September 2026
Top 25 Rare disease organizations based in the United States
RevenueBase identified 223 company matches for rare disease organizations associated with the United States as of September 2026 and tracks 13,230 professional contacts across them, including 7,640 with verified work emails. Washington is the largest hub.
This list covers company matches for rare disease organizations associated with the United States, 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.
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 based in the United States
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 | 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 | |||||
| 5 | 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 | |||||
| 6 | 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 | |||||
| 7 | 185 contacts → | ||||
Exact match Recorded description not available. Recorded in the RevenueBase company graph · Sep 21, 2026 · View company records in RevenueBase | |||||
| 8 | 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 | |||||
| 9 | 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 | |||||
| 10 | 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 | |||||
| 11 | 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 | |||||
| 12 | 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 | |||||
| 13 | 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 | |||||
| 14 | 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 | |||||
| 15 | 61 contacts → | ||||
Exact match “At Citizen Health, we have a singular mission: to improve the lives of the 400+ million people navigating rare and complex conditions.” Recorded in the RevenueBase company graph · Sep 21, 2026 · View company records in RevenueBase | |||||
| 16 | 59 contacts → | ||||
Exact match “At SCYNEXIS, we are committed to improving outcomes for patients living with severe, progressive, and underserved rare diseases.” Recorded in the RevenueBase company graph · Sep 21, 2026 · View company records in RevenueBase | |||||
| 17 | 56 contacts → | ||||
Exact match “Ask us what we did today, and we'll tell you that we worked hard to change someone's life.” Recorded in the RevenueBase company graph · Sep 21, 2026 · View company records in RevenueBase | |||||
| 18 | 46 contacts → | ||||
Exact match “Spero Therapeutics is a clinical-stage biopharmaceutical company focused on advancing next-generation medicines in immunology and inflammation for patients with serious diseases…” Recorded in the RevenueBase company graph · Sep 21, 2026 · View company records in RevenueBase | |||||
| 19 | 46 contacts → | ||||
Exact match “Accessia Health is a national nonprofit 501(c)(3) organization that provides programs and services to those living with rare or chronic health conditions.” Recorded in the RevenueBase company graph · Sep 21, 2026 · View company records in RevenueBase | |||||
| 20 | 42 contacts → | ||||
Exact match “We are passionate about bringing better treatments to people faster.” Recorded in the RevenueBase company graph · Sep 21, 2026 · View company records in RevenueBase | |||||
| 21 | 39 contacts → | ||||
Exact match “Empowering the Next Generation Rare Disease Advocate.” Recorded in the RevenueBase company graph · Sep 21, 2026 · View company records in RevenueBase | |||||
| 22 | 36 contacts → | ||||
Exact match “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…” Recorded in the RevenueBase company graph · Sep 21, 2026 · View company records in RevenueBase | |||||
| 23 | 34 contacts → | ||||
Exact match “At CareMed, we specialize in supporting patients living with rare, ultra-rare, and complex diseases by delivering the highest level of care through personalized support, clinical…” Recorded in the RevenueBase company graph · Sep 21, 2026 · View company records in RevenueBase | |||||
| 24 | 31 contacts → | ||||
Exact match “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.” Recorded in the RevenueBase company graph · Sep 21, 2026 · View company records in RevenueBase | |||||
| 25 | 21 contacts → | ||||
Exact match “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, &…” 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 223 rare disease organizations based in the United States
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Rare disease organizations 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 223 companies.
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.
- What qualifies
- Company records associated with the United States 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. Where headquarters information is recorded, preview rows are also filtered to this page’s location. 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 7,640 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.
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 based in the United States?
RevenueBase identified 223 company matches for rare disease organizations associated with the United States 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 city has the most rare disease organizations?
Washington leads with 11 company records, followed by Boston (10) and Austin (8).
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 7,640 professional contacts with verified work emails tracked across these 223 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 223 rare disease organizations based in the United States. Start with a free account, no credit card.
Download this list →Related lists
Rare disease organizations by geography
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
- All company lists
- Lists by location
- Lists A-Z
- New lists
- Rare disease organizations based in France
- Rare disease organizations based in the Netherlands
- Rare disease organizations based in the United Kingdom
- AI companies based in the United States
- Cybersecurity companies based in the United States
- Manufacturing companies based in the United States
Company intelligence · public preview
Top 25 rare disease organizations based in the United States
Find companies in any niche.
Describe the businesses you want in your own words. RevenueBase SmartSearch matches company descriptions by meaning.
RevenueBase records 223 matching company records for rare disease organizations based in the United States, with 13,230 tracked contacts and 7,640 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 based in the United States
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 |  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. | |||||
| 5 |  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. | |||||
| 6 | 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. | |||||
| 7 | 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. | |||||
| 8 | 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. | |||||
| 9 | 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. | |||||
| 10 | 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. | |||||
| 11 | 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. | |||||
| 12 |  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. | |||||
| 13 | 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. | |||||
| 14 | 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. | |||||
| 15 | Citizen Healthcitizen.health HQ: San Francisco, CA, United StatesReported employees: 11-50 | San Francisco, CA, United States | 11-50 | Exact match | 61 contacts → |
“At Citizen Health, we have a singular mission: to improve the lives of the 400+ million people navigating rare and complex conditions.” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 16 | Scynexis, INCscynexis.com HQ: Jersey City, NJ, United StatesReported employees: 11-50 | Jersey City, NJ, United States | 11-50 | Exact match | 59 contacts → |
“At SCYNEXIS, we are committed to improving outcomes for patients living with severe, progressive, and underserved rare diseases.” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 17 | Purplemoon, An Iqvia Businessgopurplemoon.com HQ: Montclair, NJ, United StatesReported employees: 51-200 | Montclair, NJ, United States | 51-200 | Exact match | 56 contacts → |
“Ask us what we did today, and we'll tell you that we worked hard to change someone's life.” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 18 | Spero Therapeuticssperotherapeutics.com HQ: Cambridge, MA, United StatesReported employees: 11-50 | Cambridge, MA, United States | 11-50 | Exact match | 46 contacts → |
“Spero Therapeutics is a clinical-stage biopharmaceutical company focused on advancing next-generation medicines in immunology and inflammation for patients with serious diseases…” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 19 |  Accessia Healthaccessiahealth.orgHQ: Midlothian, VA, United StatesReported employees: 11-50 | Midlothian, VA, United States | 11-50 | Exact match | 46 contacts → |
“Accessia Health is a national nonprofit 501(c)(3) organization that provides programs and services to those living with rare or chronic health conditions.” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 20 | Patientwingpatientwing.com HQ: Philadelphia, PA, United StatesReported employees: 51-200 | Philadelphia, PA, United States | 51-200 | Exact match | 42 contacts → |
“We are passionate about bringing better treatments to people faster.” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 21 | Global Genesglobalgenes.org HQ: Washington, United StatesReported employees: 11-50 | Washington, United States | 11-50 | Exact match | 39 contacts → |
“Empowering the Next Generation Rare Disease Advocate.” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 22 | HQ: Long Beach, CA, United StatesReported employees: 51-200 | Long Beach, CA, United States | 51-200 | Exact match | 36 contacts → |
“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…” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 23 | Caremed Specialty Pharmacycaremed.com HQ: New Hyde Park, NY, United StatesReported employees: 51-200 | New Hyde Park, NY, United States | 51-200 | Exact match | 34 contacts → |
“At CareMed, we specialize in supporting patients living with rare, ultra-rare, and complex diseases by delivering the highest level of care through personalized support, clinical…” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 24 | Rare Genomics Instituteraregenomics.org HQ: Downey, CA, United StatesReported employees: 51-200 | Downey, CA, United States | 51-200 | Exact match | 31 contacts → |
“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.” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 25 | Indo US Organization For Rare Diseases Indousrareindousrare.org HQ: Herndon, VA, United StatesReported employees: 11-50 | Herndon, VA, United States | 11-50 | Exact match | 21 contacts → |
“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, &…” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
Showing 25 of 223 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
223 records in the displayed employee bands.
0 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 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 223 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 ↗
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 based in the United States?
The saved snapshot records 223 matching company records, 13,230 tracked contacts and 7,640 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
Rare disease organizations by geography
Cite this list
RevenueBase Data Team. Top 25 rare disease organizations based in the United States. Snapshot: September 21, 2026. https://revenuebase.ai/companies/rare-disease-organizations/united-states.
This is a selected public preview of saved company records, rather than an independently verified census. Open the source page ↗
More company lists
- All company lists
- Lists by location
- Lists A-Z
- New lists
- Rare disease organizations based in France
- Rare disease organizations based in the Netherlands
- Rare disease organizations based in the United Kingdom
- AI companies based in the United States
- Cybersecurity companies based in the United States
- Manufacturing companies based in the United States
Company intelligence · public preview
Top 25 rare disease organizations based in the United States
RevenueBase records 223 matching company records for rare disease organizations based in the United States, with 13,230 tracked contacts and 7,640 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 based in the United States
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 | 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. | |||||
| 5 | 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. | |||||
| 6 | 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. | |||||
| 7 | 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. | |||||
| 8 | 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. | |||||
| 9 | 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. | |||||
| 10 | 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. | |||||
| 11 | 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. | |||||
| 12 | 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. | |||||
| 13 | 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. | |||||
| 14 | 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. | |||||
| 15 | Citizen Healthcitizen.health HQ: San Francisco, CA, United StatesReported employees: 11-50 | San Francisco, CA, United States | 11-50 | Exact match | 61 contacts → |
“At Citizen Health, we have a singular mission: to improve the lives of the 400+ million people navigating rare and complex conditions.” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 16 | Scynexis, INCscynexis.com HQ: Jersey City, NJ, United StatesReported employees: 11-50 | Jersey City, NJ, United States | 11-50 | Exact match | 59 contacts → |
“At SCYNEXIS, we are committed to improving outcomes for patients living with severe, progressive, and underserved rare diseases.” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 17 | Purplemoon, An Iqvia Businessgopurplemoon.com HQ: Montclair, NJ, United StatesReported employees: 51-200 | Montclair, NJ, United States | 51-200 | Exact match | 56 contacts → |
“Ask us what we did today, and we'll tell you that we worked hard to change someone's life.” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 18 | Spero Therapeuticssperotherapeutics.com HQ: Cambridge, MA, United StatesReported employees: 11-50 | Cambridge, MA, United States | 11-50 | Exact match | 46 contacts → |
“Spero Therapeutics is a clinical-stage biopharmaceutical company focused on advancing next-generation medicines in immunology and inflammation for patients with serious diseases…” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 19 | Accessia Healthaccessiahealth.org HQ: Midlothian, VA, United StatesReported employees: 11-50 | Midlothian, VA, United States | 11-50 | Exact match | 46 contacts → |
“Accessia Health is a national nonprofit 501(c)(3) organization that provides programs and services to those living with rare or chronic health conditions.” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 20 | Patientwingpatientwing.com HQ: Philadelphia, PA, United StatesReported employees: 51-200 | Philadelphia, PA, United States | 51-200 | Exact match | 42 contacts → |
“We are passionate about bringing better treatments to people faster.” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 21 | Global Genesglobalgenes.org HQ: Washington, United StatesReported employees: 11-50 | Washington, United States | 11-50 | Exact match | 39 contacts → |
“Empowering the Next Generation Rare Disease Advocate.” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 22 | HQ: Long Beach, CA, United StatesReported employees: 51-200 | Long Beach, CA, United States | 51-200 | Exact match | 36 contacts → |
“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…” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 23 | Caremed Specialty Pharmacycaremed.com HQ: New Hyde Park, NY, United StatesReported employees: 51-200 | New Hyde Park, NY, United States | 51-200 | Exact match | 34 contacts → |
“At CareMed, we specialize in supporting patients living with rare, ultra-rare, and complex diseases by delivering the highest level of care through personalized support, clinical…” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 24 | Rare Genomics Instituteraregenomics.org HQ: Downey, CA, United StatesReported employees: 51-200 | Downey, CA, United States | 51-200 | Exact match | 31 contacts → |
“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.” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 25 | Indo US Organization For Rare Diseases Indousrareindousrare.org HQ: Herndon, VA, United StatesReported employees: 11-50 | Herndon, VA, United States | 11-50 | Exact match | 21 contacts → |
“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, &…” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
Showing 25 of 223 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
223 records in the displayed employee bands.
0 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 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 223 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 ↗
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 based in the United States?
The saved snapshot records 223 matching company records, 13,230 tracked contacts and 7,640 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
Rare disease organizations by geography
Cite this list
RevenueBase Data Team. Top 25 rare disease organizations based in the United States. Snapshot: September 21, 2026. https://revenuebase.ai/companies/rare-disease-organizations/united-states.
This is a selected public preview of saved company records, rather than an independently verified census. Open the source page ↗
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