National Gaucher Foundation

What National Gaucher Foundation does

The National Gaucher Foundation (NGF) is a 501(c)(3) nonprofit patient advocacy organization headquartered in Rockville, Maryland, dedicated to serving individuals with Gaucher disease — a rare inherited lysosomal storage disorder affecting up to 6,000 people in the United States. Founded with Brian Berman as CEO since January 2016 (himself the first person in the world to successfully receive enzyme replacement therapy for Gaucher disease), NGF operates a community-led go-to-market model reaching patients and families through its website, blog, email newsletter (Gaucher Today), social media, webinars, in-person meetings, and an elite Ambassador Program. Core programmatic offerings include the CARE Program (insurance premium assistance), Care+Plus (out-of-pocket medical expense support), the Optimal Health Initiative, a Treatment Finder specialist locator, a Knowledge Center, and CME-accredited physician education modules.

The organization does not develop proprietary technology products; its infrastructure consists of a content-driven website and conventional digital channels. NGF monetizes its mission through tax-deductible charitable donations, a recurring-gift Stepping Stones sustainer program, and likely pharmaceutical-company partnerships that connect patients with enzyme replacement therapy and substrate reduction therapy manufacturers. Strategic partnerships extend its reach: JScreen (Emory University) for genetic carrier screening, Mayo Clinic for confirmatory diagnostic testing, Greenwood Genetics Center for sibling-focused diagnostics, and a Global Diagnostic and Treatment Initiative that has produced the first diagnosed Gaucher disease case in Zambia (2024) and is expanding to India. The organization is supported by a 6-member Medical Advisory Board of leading Gaucher specialists and works alongside its affiliated nonprofit, National Gaucher CARE Foundation, Inc. (EIN 52-1815708). Revenue is not publicly disclosed; the foundation operates with a small staff (three named personnel) and is not a dividend- or exit-driven entity.

Name

National Gaucher Foundation

Legal name

National Gaucher Foundation, Inc.

Website

https://gaucherdisease.org

Company type

Private

Founded year

1984

Operating status

Operating

Headcount range

1–10 employees

Short description

The National Gaucher Foundation is a 501(c)(3) patient advocacy nonprofit that supports the estimated 6,000 U.S. individuals with Gaucher disease through financial assistance (CARE Program, Care+Plus), education, specialist referrals, ambassador programs, and a Global Diagnostic Treatment Initiative, funded by donations and pharmaceutical partnerships.

Ownership category

akta.pro rank

Product category

Rare Disease Patient Advocacy

NAICS

Voluntary Health Organizations (813212), Grantmaking and Giving Services (81321)

SIC

Services-Social Services (8300)

akta.pro primary industry

Public Health Financing, Budgeting & Grants Management (HLAJAMAC)

Keywords

• Rare disease advocacy
• Patient support services
• Genetic disease education
• Financial assistance programs
• Nonprofit health services

Headquarters

HQ city

Rockville

HQ country

United States

HQ region

North America

Offices1 record

Markets served

GTM type

B2C

Offering type

Services

Cost components

Personnel, Operations, Marketing or Sales, Others, Technology or R&D

Revenue model

1. Charitable Donations: The organization is a 501(c)(3) nonprofit exempt from federal income tax. Donations are tax-deductible to the extent permitted by law. The foundation receives support from individual donors and likely pharmaceutical company partnerships.
2. Patient Financial Assistance Programs: NGF offers financial support programs including the CARE Program (helps with insurance premiums) and Care+Plus (helps with certain out-of-pocket expenses) to Gaucher patients and their families.

Go-to-market motion1 record

Distribution channels4 records

Marketing channels8 records

Core offering

National Gaucher Foundation is a 501(c)(3) nonprofit patient advocacy organization that supports individuals and families affected by Gaucher disease through financial assistance programs (CARE Program, Care+Plus), educational programming, patient services such as a Treatment Finder, an Optimal Health Initiative, and the NGF Global Diagnostic and Treatment Initiative. It also runs an NGF Ambassador peer-support program and provides physician resources including CME-accredited educational modules.

Product overview

The National Gaucher Foundation is a 501(c)(3) nonprofit patient advocacy organization, not a technology product company. Its offerings consist of patient support programs and services rather than a unified software product or platform. The core services include the CARE Program and Care+Plus (financial assistance), the Optimal Health Initiative (specialist engagement), the Global Diagnostic and Treatment Initiative (international diagnosis support), the NGF Stepping Stones sustainer program (donations), the Treatment Finder tool, the Knowledge Center/Blog (educational content), the NGF Ambassador Program (patient advocacy), and Physician Resources with CME programs. These programs are accessed through the organization's main website and are designed to support U.S. patients with Gaucher disease through financial support, educational programming, patient services, and collaboration with medical professionals.

Differentiator

Problem solved

Functional benefit

Products and services

• CARE Program
• Care+Plus Program Financial assistance program that helps Gaucher patients with certain out-of-pocket medical expenses related to their care.
• Optimal Health Initiative (OHI) Program that encourages Gaucher patients to engage with specialists and adopt a proactive approach to managing their health, including patient-focused team support across age groups.
• NGF Global Diagnostic and Treatment Initiative International program helping diagnose and support people with Gaucher disease in resource-limited countries. NGF covers BGL (beta-glucosidase leukocyte) testing costs and coordinates with pharmaceutical companies to provide enzyme replacement therapy at no charge.
• Treatment Finder Online tool that helps patients locate Gaucher disease specialists and treatment centers across the United States.
• NGF Ambassador Program Elite peer-support program that connects trained patient advocates living with Gaucher disease with newly diagnosed patients and the broader community for guidance and shared experience.
• Physician Resources and CME Programs CME-accredited educational modules and digital resources that help hematologists, geneticists, pediatricians, and other physicians recognize Gaucher disease symptoms and connect patients with specialists.
• NGF Stepping Stones Sustainer Program Recurring donation program that enables supporters to make an ongoing financial impact supporting people living with Gaucher disease across the country.

Quantifiable outcome

• Reduced diagnostic delays through education campaigns
• +2 more outcomes

Named customers5 records

Segments3 records

Ideal customer profiles4 records

Technology focussed No

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Partnerships

Six partnerships are on record, tiered minor and core.

• European Gaucher AllianceminorOthers · 1 January 2026NGF's privacy policy recommends that individuals residing within the EU contact the European Gaucher Alliance organization operating in Europe for information about Gaucher disease and local support resources.
• Pharmaceutical Companies (ERT Manufacturers)coreGTM or Marketing Partner · 1 January 2024NGF connects patients diagnosed through the Global Diagnostic Treatment Initiative with pharmaceutical companies that provide enzyme replacement therapy (ERT). Dr. Chirwa applies to pharmaceutical companies on behalf of patients, who receive ERT free of charge. The entire application process takes about 6 months from application to receiving medicine in-country.
• JScreen (Emory University School of Medicine)coreStrategic or Co-development PartnerNGF partners with JScreen, a community-based public health initiative from Emory University School of Medicine, to provide genetic carrier screening for Gaucher disease and over 200 other genetic diseases. The partnership offers at-home saliva-based test kits. NGF sponsors screening initiatives to help reduce the cost burden for at-risk populations. Genetic counseling is included with results.
• Greenwood Genetics CenterminorStrategic or Co-development PartnerNGF partnered with Greenwood Genetics Center for a diagnostic initiative specifically for people who have siblings with Gaucher disease and have yet to have genetic testing. A simple blood test is used for this testing.
• National Gaucher CARE Foundation, Inc.coreOthersNGF works in conjunction with its affiliate National Gaucher CARE Foundation, Inc. Both organizations are exempt from federal income tax pursuant to Section 501(c)(3) of the Internal Revenue Code. National Gaucher Foundation, Inc.'s employer identification number is 52-1350226. National Gaucher CARE Foundation, Inc.'s employer identification number is 52-1815708. Both are incorporated in the State of Maryland.
• Mayo ClinicminorStrategic or Co-development PartnerNGF sends patient blood samples to Mayo Clinic for confirmatory diagnostic testing when initial testing indicates possible Gaucher disease. Mayo Clinic serves as a reference laboratory for the foundation's diagnostic initiatives.

Scale indicators2 records

Recent moves6 records

Expansion highlights5 records

Direct peers

• Genetic Alliance: U.S. nonprofit advocacy coalition for individuals and families affected by genetic conditions. Comparable in patient navigation, screening partnerships, and policy advocacy for genetic disease communities.
• National MPS Society: U.S. advocacy organization for mucopolysaccharidoses (MPS) and related lysosomal storage diseases. Directly comparable in ultra-rare genetic disease advocacy, family support, and treatment access programs.
• Acid Maltase Deficiency Association (AMDA): Patient advocacy for Pompe disease (another lysosomal storage disorder). Closely comparable in disease rarity, patient-assistance model, and pharma partnerships for enzyme replacement.
• Cystic Fibrosis Foundation: Disease-specific patient advocacy and research nonprofit with a much larger scale. Directly comparable in mission, patient support programs, pharma-funded research, and Treatment Center model.
• Muscular Dystrophy Association: Disease-specific rare disease nonprofit providing patient services, research funding, and care-center networks. Directly comparable in patient-assistance, advocacy, and clinical education programs.
• EveryLife Foundation for Rare Diseases: U.S. rare disease advocacy nonprofit focused on policy, patient engagement, and newborn screening. Highly comparable in advocacy mission, donor-funded nonprofit model, and patient community focus.
• National Hemophilia Foundation: U.S. patient advocacy organization for hemophilia and inherited blood disorders. Closely comparable model—genetic hematologic condition, patient financial assistance, community programs, and CME education for providers.
• Fabry Disease patient organizations (e.g., Fabry Support & Information Group): Patient advocacy for Fabry disease—another lysosomal storage disorder closely related to Gaucher. Highly comparable in disease archetype, patient population size, treatment costs, and diagnostic-delay challenges.

Broad incumbents

• National Organization for Rare Disorders (NORD): The largest U.S. umbrella advocacy organization for rare diseases. Directly comparable in patient-advocacy, education, and financial-assistance model, but covers 7,000+ diseases rather than being dedicated to Gaucher.

Regional players

• European Gaucher Alliance: Pan-European federation of Gaucher disease patient associations. Directly comparable to NGF in disease focus and patient-advocacy mission, but operates across European geographies rather than the U.S. NGF already refers EU residents to this organization.

Market position

Strengths4 records

Weaknesses4 records

Competitive moat5 records

Key risks4 records

Key highlights6 records

Customer concentration

• Facebookfacebook.com/nationalgaucherfoundation
• LinkedInlinkedin.com/company/national-gaucher-foundation
• Instagraminstagram.com/gaucherdisease

Revenue estimate

Valuation estimate

Number of profiles

Profiles4 records

Funding overview

Funding rounds

Investors

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M&A

Investments

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Frequently asked questions about National Gaucher Foundation