National Niemann-Pick disease Foundation

What National Niemann-Pick disease Foundation does

The National Niemann-Pick Disease Foundation (NNPDF) is a 501(c)(3) non-profit patient advocacy organization founded in 1992 (incorporated and renamed in 1993) and headquartered in Fort Atkinson, Wisconsin. It serves over 450 member families across the United States affected by Niemann-Pick disease, specifically ASMD (Types A and B) and NPC (Type C). The organization's portfolio of services includes the Family Assistance and Support Program (FASP) providing up to $1,000 per year in financial relief per eligible family, one-on-one Family Support Services for crisis and emotional support, a Bereavement Program with licensed grief counselors, Community Connections virtual chats, and the Bringing Holiday Cheer holiday assistance initiative. NNPDF co-founded the International Niemann-Pick Disease Registry (INPDR), a global patient registry for clinical and genetic data, and co-founded the International Niemann-Pick Disease Alliance (INPDA) in 2009 alongside the Lysosomal Disease Research Consortium, which together award over $1 million in research grants annually. The foundation maintains a Scientific Advisory Board and operates the largest annual Family Support & Medical Conference in the world for the disease (33rd edition in July 2025), which has grown approximately 15-fold since inception and now runs parallel ASMD and NPC tracks. NNPDF does not develop technology products; its platform consists of standard non-profit management tools, CRM for member engagement, and virtual meeting infrastructure.

NNPDF is funded entirely through donations, contributions, and pharmaceutical industry partnerships. Named industry partners include Azafaros, Beren Therapeutics, Cyclo Therapeutics, IntraBio, Sanofi (a core, mutual relationship), and Zevra Therapeutics, several of which have recently secured FDA approvals for Niemann-Pick treatments (AQNEURSA by IntraBio and MIPLYFFA by Zevra). Membership is complimentary, and the organization reaches families through a multi-channel strategy including its website (nnpdf.org), a newsletter distributed to over 1,000 households, social media (Facebook, Instagram, LinkedIn, Twitter/X), regional family events, annual conferences, and partnerships with healthcare providers. The organization operates with three core paid staff (Executive Director, Communications & Marketing Coordinator, Family Services Manager) supported by an unpaid volunteer Board of Directors, the majority of whom are parents of affected children.

Name

National Niemann-Pick disease Foundation

Legal name

National Niemann-Pick Disease Foundation, Inc.

Website

https://nnpdf.org

Company type

Private

Founded year

1993

Operating status

Operating

Headcount range

1–10 employees

Short description

The National Niemann-Pick Disease Foundation is a 501(c)(3) non-profit founded in 1993 that supports over 450 U.S. families affected by Niemann-Pick disease (ASMD and NPC) through family support services, financial assistance, research funding, advocacy, and the world's largest annual family conference for the disease.

Ownership category

akta.pro rank

Headquarters

HQ city

Fort Atkinson

HQ country

United States

HQ region

North America

Offices1 record

Markets served

GTM type

B2C

Offering type

Services

Cost components

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

Revenue model

1. Donations and Contributions: As a 501(c)(3) non-profit organization, NNPDF generates revenue primarily through donations from individuals, families, and corporate partners. Contributions support research funding, family services programs, and operational costs. The organization receives funding from pharmaceutical industry partners including Azafaros, Beren Therapeutics, Cyclo Therapeutics, IntraBio, Sanofi, and Zevra. Donations are tax-deductible as allowed by law (Tax ID# 35-1844264).

Go-to-market motion1 record

Distribution channels5 records

Marketing channels14 records

Core offering

NNPDF is a 501(c)(3) non-profit patient advocacy and family support organization serving U.S. families affected by Niemann-Pick disease (ASMD Types A/B and NPC). It delivers free family membership, emotional and crisis support, the Family Assistance and Support Program (FASP) providing up to $1,000 annually per eligible family, advocacy at federal and state levels, annual conferences and regional events, and funds research through the Lysosomal Disease Research Consortium. The foundation co-founded the International Niemann-Pick Disease Alliance (INPDA) and supports the global INPDR disease registry.

Product overview

The National Niemann-Pick Disease Foundation (NNPDF) is a non-profit patient advocacy and family support organization, not a technology product company. NNPDF provides a coordinated portfolio of support services and programs for families affected by Niemann-Pick disease types ASMD and NPC. Core offerings include the Family Assistance and Support Program (FASP) providing financial relief up to $1,000 annually, Family Support Services offering emotional and crisis support, the annual Family Support & Medical Conference held since 1992, advocacy programs at federal/state levels, Community Connections Chats, Bereavement Program, and the Bringing Holiday Cheer program. Through its subsidiary INPDR, the foundation supports a global patient registry for Niemann-Pick disease research.

Differentiator

Problem solved

Functional benefit

Products and services

• Family Assistance and Support Program (FASP) Provides short-term financial assistance of up to $1,000 per 12-month period for eligible NNPDF U.S. member families facing hardship, covering specialized medical equipment, utility bills, home/car repairs, and bereavement expenses.
• Family Support Services Emotional support, crisis assistance, and information sharing for families dealing with Niemann-Pick disease, including connections to medical experts, diagnostic testing, treatment options, and peer support from experienced families.
• Annual Family Support & Medical Conference Annual conference bringing together families, scientists, and medical professionals to share information and provide support, with separate sessions for ASMD and NPC disease types.
• Advocacy Program Advocacy initiatives at federal, state, and regional levels, including newborn screening advocacy, FDA engagement, and collaboration with NORD and the Rare & Ready Genetic Condition Coalition.
• Community Connections Chats Virtual Zoom meetings allowing community members to connect, plus the Navigating Together series providing confidential small group sessions led by licensed grief counselors.
• Bereavement Program Emotional support for families experiencing loss, including a series of booklets during the first year, memorial tributes on social media, and Navigating Together: Legacy Families small group sessions.
• Bringing Holiday Cheer Program Holiday assistance program providing wish list items shipped to eligible NNPDF U.S. member families to brighten the holiday season for families affected by Niemann-Pick disease.
• International Niemann-Pick Disease Registry (INPDR) Global disease registry collecting clinical, genetic, diagnostic, and outcome data from patients with all types of Niemann-Pick disease to support research and improve clinical care worldwide.

Named customers1 record

Segments4 records

Ideal customer profiles3 records

Technology focussed No

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Partnerships

Twelve partnerships are on record, tiered minor and core.

• AzafarosminorStrategic or Co-development PartnerPharmaceutical company developing treatments for rare diseases including Niemann-Pick disease. Listed as a funding partner supporting NNPDF's mission and the Niemann-Pick community. At 2025 conference, presented clinical update on nizubaglustat for NPC.
• Beren TherapeuticsminorStrategic or Co-development PartnerBiotechnology company focused on rare disease therapeutics. Listed as a funding partner supporting NNPDF's mission.
• Cyclo TherapeuticsminorStrategic or Co-development PartnerCompany developing Trappsol Cyclo for NPC. Listed as a funding partner. At 2025 conference, presented clinical update on Trappsol Cyclo. Received Breakthrough Award consideration for their NPC treatment work.
• IntraBiominorStrategic or Co-development PartnerPharmaceutical company that received FDA approval for AQNEURSA (N-acetyl-L-leucine) for NPC. Listed as a funding partner. 2025 Breakthrough Award recipient. Presented at 2025 conference on approved treatments.
• SanoficoreStrategic or Co-development PartnerMajor pharmaceutical partner developing olipudase alfa for ASMD (approved as Xenpapzyme in some regions). Provides patient support services. 2023 Breakthrough Award recipient. Co-sponsored first International Symposium on NPC. Active in clinical trials and treatment development.
• Zevra TherapeuticsminorStrategic or Co-development PartnerCompany that received FDA approval for MIPLYFFA (migalastat) for NPC. Listed as a funding partner. 2025 Breakthrough Award recipient. Presented at 2025 conference on approved treatments.
• International Niemann-Pick Disease Alliance (INPDA)coreStrategic or Co-development PartnerGlobal network of non-profit organizations supporting persons affected by Niemann-Pick diseases. NNPDF is the US member organization. Formed in 2009 to provide forum for patient groups and professionals. INPDA Executive Committee includes President Sandy Cowie (Canada), Vice President Joslyn Crowe (USA), and others. Provides mutual support, shares expertise and information, and stimulates research internationally.
• Lysosomal Disease Research ConsortiumcoreStrategic or Co-development PartnerNNPDF is a founding member of this consortium which awards over $1,000,000 in related research grants annually to advance treatments for lysosomal storage disorders including Niemann-Pick disease.
• International Niemann-Pick Disease Registry (INPDR)coreStrategic or Co-development PartnerA global disease-specific registry, a subsidiary of INPDA, collecting clinical, genetic, diagnostic, and outcome data from patients with all types of Niemann-Pick disease. Collaboration between clinicians, scientists, researchers, and patient associations worldwide. Based in UK as a not-for-profit company.
• The Assistance FundminorStrategic or Co-development PartnerIndependent non-profit organization providing financial assistance for out-of-pocket costs associated with FDA-approved ASMD treatments. Program covers prescription drug assistance, health insurance premiums, therapy administration costs, disease management, treatment-related travel costs, and genetic testing. Helps over 160,000 children and adults since founding in 2009.
• National Organization for Rare Disorders (NORD)minorStrategic or Co-development PartnerNNPDF works with NORD on advocacy initiatives including Rare Disease Advisory Councils and other rare disease policy efforts at state and federal levels.
• Rare & Ready Genetic Condition CoalitionminorStrategic or Co-development PartnerStatewide grassroots advocacy coalition of 46 non-profit rare disease organizations. NNPDF is a partner working to improve timely access to FDA-approved medicines in Medicaid for those with rare and genetic conditions.

Scale indicators9 records

Recent moves6 records

Expansion highlights5 records

Direct peers

• National Organization for Rare Disorders (NORD): NORD is the umbrella U.S. rare-disease advocacy organization and a direct peer to NNPDF: both engage in federal/state policy advocacy (Rare Disease Week, Rare Disease Advisory Councils), partner with pharma on drug development, and serve as connectors between disease-specific foundations and broader rare-disease infrastructure. NNPDF explicitly collaborates with NORD.
• American Migraine Foundation: American Migraine Foundation is a patient advocacy and education organization with an analogous operating model: family/patient education, clinician collaboration, research funding, and a small professional staff. NNPDF's Executive Director previously served as Director of Programs & Advocacy there, signaling direct operational lineage.
• Acid Maltase Deficiency Association (Pompe): AMDA is a patient advocacy organization for Pompe disease (a lysosomal storage disorder closely related to Niemann-Pick), making it a tightly comparable peer. Both serve small populations with lysosomal storage diseases, partner with biotech on enzyme replacement/gene therapy programs, and operate family-support and research-funding models.
• International Niemann-Pick Disease Alliance (INPDA): INPDA is the global umbrella organization co-founded by NNPDF in 2009, comprising Niemann-Pick patient organizations worldwide. It is the most direct peer in mission, though at a coordinating rather than direct-service level.
• Parent Project Muscular Dystrophy: PPMD is a parent-founded rare-disease advocacy nonprofit that funds Duchenne muscular dystrophy research, runs family conferences, and engages pharma on clinical trial recruitment — directly analogous to NNPDF's structure for an ultra-rare pediatric population.
• Batten Disease Support, Research, and Advocacy Foundation: BDSRA is an ultra-rare disease family foundation with an operating model highly comparable to NNPDF: small staff, volunteer family-led board, family conferences, research funding, pharma partnerships for an ultra-rare pediatric neurodegenerative disease. It is one of the closest structural analogs in the rare-disease nonprofit ecosystem.

Broad incumbents

• ALS Association: The ALS Association is a large U.S. patient advocacy nonprofit for a fatal neurodegenerative disease, comparable in mission structure: family services, research grants, federal advocacy, annual conferences, and pharma engagement. Like NNPDF, it serves families navigating a progressive disease with limited treatment options.
• Muscular Dystrophy Association: MDA is a long-established rare-disease advocacy organization funding research, providing family support, and partnering with pharma across multiple neuromuscular diseases. Its multi-disease portfolio is broader than NNPDF's, but the core operating model — research funding, clinical care centers, family services, advocacy — is closely comparable.
• National Multiple Sclerosis Society: The National MS Society is a major patient advocacy and research-funding nonprofit serving people with a chronic neurological disease. It shares NNPDF's DNA of family/patient education, research grants, federal advocacy, pharma partnerships, and an annual conference structure, though at significantly larger scale.
• Cystic Fibrosis Foundation: The Cystic Fibrosis Foundation is a leading U.S. rare-disease patient advocacy organization with a comparable model: family support services, research funding via an affiliated research foundation, pharma partnerships, an annual family conference, and a volunteer board largely composed of affected families. NNPDF's Family Services Manager previously worked at CFF for 10+ years, indicating operational similarity.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat5 records

Key risks5 records

Key highlights7 records

Customer concentration

• Xx.com/nnpdf
• Facebookfacebook.com/NNPDF
• LinkedInlinkedin.com/company/national-niemann-pick-disease-foundation
• Instagraminstagram.com/nnpdf

Revenue estimate

Valuation estimate

Number of profiles

Profiles3 records

Funding overview

Funding rounds

Investors

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M&A

Investments

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