What Hope for PDCD Foundation does

Hope for PDCD Foundation is a 501(c)(3) nonprofit patient advocacy organization founded in 2022 and headquartered in Folsom, California, dedicated to finding a cure for Pyruvate Dehydrogenase Complex Deficiency (PDCD), a rare mitochondrial disorder affecting roughly 1 in 40,000 live births (approximately 90 US newborns annually). The foundation is completely parent-powered and volunteer-run with five employees and a stated 100% pass-through of donations to research, advocacy, and community programs; it does not pay overhead or salaries.

Its core programmatic surface spans four research pillars: AAV9 gene replacement therapy for the PDHA1 gene (developed in partnership with the Gray Lab at UT Southwestern, with Stage 1 mice model work completed in 2023 and Stage 2 toxicology/GLP safety studies underway), small molecule therapies including dichloroacetate (DCA) and triheptanoin (C7), a PDCD Natural History Study at UPMC Children's Hospital of Pittsburgh enrolling 150+ subjects across the US and Canada, and advocacy for adding PDCD to the Recommended Uniform Screening Panel (RUSP). It also operates an international patient registry on Sanford Research's CoRDS platform and a community/research portal with clinician directories, free genetic testing resources, and standards-of-care work.

The foundation's revenue model is multi-stream and donation-centric: individual donations (one-time and monthly), corporate matching gifts through Benevity (700+ partner companies), tiered corporate sponsorships from $500 (Fish Friends) to $25,000+ (MCT Oil Platinum), merchandise sales ($4.50–$39 items), Facebook birthday fundraisers, and an affiliate partnership with Minted. Customer segments are primarily PDCD patient families (parents, caretakers, extended family) and secondarily rare disease medical researchers and mitochondrial disease clinicians; marketing is community-led via organic social, peer-to-peer campaigns, the annual Butter Challenge, and FDA advocacy events.

Name

Hope for PDCD Foundation

Legal name

Hope for PDCD Foundation

Website

https://hopeforpdcd.org

Company type

Private

Operating status

Operating

Headcount range

1–10 employees

Short description

Hope for PDCD Foundation is a parent-powered, volunteer-run 501(c)(3) nonprofit that funds research, advocacy, and community programs for Pyruvate Dehydrogenase Complex Deficiency, a rare mitochondrial disease affecting roughly 90 US newborns annually.

Ownership category

akta.pro rank

Product category

Rare Disease Patient Advocacy Nonprofit

NAICS

Voluntary Health Organizations (813212), Scientific Research and Development Services (5417)

SIC

Services-Commercial Physical & Biological Research (8731)

akta.pro primary industry

Disease-Specific Research & Support (e.g., Cancer, Diabetes, ALS) (BPAGACAA)

akta.pro secondary industry

Global Health Research, Clinical Trials & Product Development Partnerships (PDPs) (HLAJAOAM)

Keywords

• Rare disease research
• Patient advocacy nonprofit
• Gene therapy funding
• Mitochondrial disease foundation
• Community fundraising campaigns

Headquarters

HQ city

Folsom

HQ country

United States

HQ region

North America

Offices1 record

Markets served

GTM type

B2C

Offering type

Services

Cost components

Technology or R&D, Marketing or Sales, Operations

Revenue model

1. Individual Donations: Tax-deductible donations from individuals supporting PDCD research. Includes one-time gifts and monthly recurring donations.
2. Corporate Matching Gifts: Part of Benevity Community Impact Fund, partnering with 700+ companies to match employee donations.
3. Sponsorship Programs: Tiered corporate sponsorship levels from $500 (Fish Friends) to $25,000+ (MCT Oil Platinum), providing visibility benefits including logo placement, event tickets, and social media recognition.
4. Merchandise Sales: Online store selling bracelets, t-shirts, stickers, and other items. All profits fund research. Price points range from $4.50 stickers to $39 t-shirts.
5. Facebook Fundraising: Facebook birthday fundraisers where the platform covers all transaction fees for donations to the nonprofit.
6. Minted Partnership: Minted stationery partner donating 15% of purchases back to Hope for PDCD when using code FUNDRAISEPDCD.

Pricing tiers

Go-to-market motion1 record

Distribution channels6 records

Marketing channels9 records

Core offering

Hope for PDCD Foundation is a 501(c)(3) nonprofit that funds and advocates for research to cure Pyruvate Dehydrogenase Complex Deficiency (PDCD), a rare mitochondrial disorder. It directs donations to AAV9 gene therapy research at the UTSW Gray Lab, small molecule therapy work (DCA and triheptanoin), newborn screening advocacy, and natural history studies, while operating an awareness merchandise store and a patient registry on the CoRDS platform.

Product overview

Hope for PDCD Foundation is a 501(c)(3) nonprofit patient advocacy organization that does not operate a unified software product but rather offers a portfolio of programs and services to advance research for Pyruvate Dehydrogenase Complex Deficiency (PDCD). The core offerings include: the PDCD Patient Registry (a data collection platform hosted on CoRDS for patient enrollment), the PDCD Natural History Study (clinical research with UPMC), and AAV9 Gene Therapy Research initiatives with UTSW's Gray Lab. The foundation also operates a merchandise store featuring awareness items (bracelets, stickers, t-shirts including 'Powerful and Rare Unicorn' and 'Keto Does It Butter' designs) with proceeds funding research. Additional programs include the Butter Challenge fundraising campaign, Small Molecule Therapy Research advocacy, and Newborn Screening advocacy efforts.

Differentiator

Problem solved

Functional benefit

Products and services

• Disability Pride Sticker A 4x4 inch vinyl sticker featuring disability pride messaging, sold online with 100% of profits funding PDCD research.
• Powerful and Rare Unicorn Sticker A 4x4 inch awareness sticker featuring a unicorn design and 'Powerful and Rare' messaging to raise awareness for PDCD.
• Hope for PDCD Sticker A branded sticker featuring the Hope for PDCD design to raise awareness and show support for the cause.
• Care About Rare Sticker An awareness sticker featuring rare disease pride messaging to promote awareness for rare diseases including PDCD.
• Listen to Parents Sticker An awareness sticker reminding healthcare providers to listen to rare disease parents, available in 3x3 or 4x4 inch sizes.
• Hope for PDCD Gradient Sticker A 90s-inspired gradient design sticker featuring the Hope for PDCD branding to raise awareness.
• Keto Does It Butter Tee (Unisex Adult) A unisex adult t-shirt featuring the 'Keto Does It Butter' slogan, screen printed on 100% organic ring-spun cotton, available in sizes XS to 5XL. Designed by Psychic Friends Club for Hope for PDCD.
• Keto Does It Butter Tee (Youth) A youth-sized t-shirt featuring the 'Keto Does It Butter' slogan, part of the ketogenic diet awareness merchandise line.
• Keto Does It Butter Tee (Toddler) A toddler-sized t-shirt featuring the 'Keto Does It Butter' slogan, screen printed on 100% organic ring-spun cotton, available in sizes 2T-5T.
• Hope for PDCD Bracelet A bracelet merchandise item sold online with 100% of profits funding PDCD research and life-saving treatments.
• Powerful and Rare Unicorn Tee (Toddler) A toddler-sized t-shirt featuring the 'Powerful and Rare Unicorn' design to raise awareness for PDCD.
• Powerful and Rare Unicorn Tee (Youth) A youth-sized t-shirt featuring the 'Powerful and Rare Unicorn' design to raise PDCD awareness.
• Powerful and Rare Unicorn Tee (Men's) A men's-sized t-shirt featuring the 'Powerful and Rare Unicorn' design to raise PDCD awareness.
• PDCD Patient Registry An international patient registry hosted on the CoRDS platform by Sanford Research that collects patient diagnosis, demographics, symptoms, healthcare utilization, diet modifications, and caregiver burden information to advance PDCD research and clinical trials.
• PDCD Natural History Study A research study conducted by UPMC Children's Hospital of Pittsburgh collecting medical records and biological samples to understand disease progression and support future investigational studies and therapies for PDCD.

Quantifiable outcome

• Successfully completed Stage 1: Mice Model Study after raising $500,000 in 2023
• +2 more outcomes

Named customers2 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

Feature4 records

Partnerships

Nine partnerships are on record, tiered core, major and minor.

• Gray Lab at UT Southwestern Medical CentercoreStrategic or Co-development Partner · 1 November 2022Primary research partner conducting PDHA1 gene therapy proof-of-concept mice model study. Successfully completed Phase 1 in 2023 after raising $500,000. Stage 2 funding going toward manufacturing AAV toxicology batch and GLP safety studies.
• Elizabeth Watt PDCD Research FundcoreStrategic or Co-development PartnerAcknowledged as the first patient advocacy group solely dedicated to PDCD in the US. Their dedication and efforts over the last decade laid the groundwork for the foundation of PDCD research and they continue to fund novel treatments for PDCD.
• Sanford Research (CoRDS Platform)coreTechnology or IntegrationHosts the international PDCD patient registry on their CoRDS platform. Strategic decision to reduce burden on 25-35% of PDCD patients who also have Leigh Syndrome diagnosis, allowing single-platform registration.
• Saol TherapeuticscoreStrategic or Co-development PartnerSponsor of Phase III clinical trial for Dichloroacetate (DCA) for PDCD treatment. On August 27, 2025, FDA issued Complete Response Letter denying full approval. Hope for PDCD organizing petition with 17,700+ signatures urging path forward.
• Children's Hospital of Philadelphia (CHOP)majorStrategic or Co-development PartnerPartner for new diagnostic tool development. Dr. Rebecca Ganetzky serves as Director of Biochemical Test Development. Also involved in newborn screening advocacy and standards of care research.
• UPMC Children's Hospital of PittsburghmajorStrategic or Co-development PartnerLeading PDCD Natural History Study enrolling minimum 150 subjects from medical centers in USA and Canada. Dr. Jirair Bedoyan's team conducting comprehensive disease progression research.
• Benevity Community Impact FundminorGTM or Marketing PartnerBenevity partners with 700+ companies to match employee donations to Hope for PDCD. Part of organization's corporate matching gift program.
• MintedminorGTM or Marketing PartnerStationery partner offering 20% discount and donating 15% of purchases to Hope for PDCD using code FUNDRAISEPDCD.
• Rep. Rutherford (FL)minorGTM or Marketing PartnerCongressman advocating for PDCD screening and research funding during World Mito Awareness Week.

Scale indicators9 records

Recent moves6 records

Expansion highlights5 records

Broad incumbents

• Cure SMA: Established rare disease nonprofit that drove the approval of the first SMA gene therapy (Zolgensma) using AAV9. Highly comparable model across fundraising, FDA advocacy, and gene therapy research funding, now operating at a much larger scale.
• United Mitochondrial Disease Foundation (UMDF): The largest US advocacy and research funding organization for mitochondrial diseases, including PDCD. Operates a much broader portfolio of programs (research grants, patient support, symposia) and serves as the umbrella organization of which Hope for PDCD is a disease-specific affiliate.
• MitoAction: A US-based nonprofit providing education, advocacy, and patient support across the mitochondrial disease spectrum, including PDCD. Comparable patient-family community model and disease awareness mission, broader in scope.
• National Organization for Rare Disorders (NORD): The umbrella advocacy organization for all rare diseases in the US. Provides grant programs, policy advocacy, and patient support infrastructure that disease-specific groups like Hope for PDCD interface with and leverage for federal policy and newborn screening initiatives.

Emerging players

• Cure SPG50: Parent-led nonprofit that funded an AAV9 gene therapy for SPG50, now in clinical trials. Closely comparable operating model: parent-driven fundraising, academic research partnership, and AAV9-based therapeutic strategy for a rare pediatric neurological disease.
• Batten Disease Support and Research Association (BDSRA): Family-led advocacy organization that helped drive the CLN7 Batten disease gene therapy program, a model Hope for PDCD explicitly cites. Similar parent-powered, gene-therapy-focused approach for an ultra-rare pediatric neurodegenerative disease.

Others

• Haystack Project: A US-based nonprofit coalition focused on ultra-rare disease policy and reimbursement advocacy. Adjacent to Hope for PDCD's mission in that it provides shared advocacy infrastructure for rare disease patient groups, though it does not directly fund disease research.

Regional players

• The Lily Foundation: The UK's leading mitochondrial disease charity, funding research and supporting families. Operates the same disease-focused advocacy and research funding model as Hope for PDCD but serves a UK patient base, with occasional cross-Atlantic collaboration through NAMDC.
• Mito Foundation: Australia's peak body for mitochondrial disease, supporting patients and funding research. Highly comparable in mission and operating model to Hope for PDCD but anchored in the Australian healthcare and research ecosystem rather than the US.

Direct peers

• Elizabeth Watt PDCD Research Fund: The first patient advocacy group solely dedicated to PDCD in the US, explicitly acknowledged by Hope for PDCD as its predecessor organization. Both charities fund PDCD research and serve the same ultra-rare disease patient community, making them the closest direct peer.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat4 records

Key risks6 records

Key highlights6 records

Customer concentration

• Xx.com/hopeforpdcd
• Facebookfacebook.com/hopeforpdcd
• LinkedInlinkedin.com/company/hopeforpdcd
• Instagraminstagram.com/hopeforpdcd

Compliance1 record

Revenue estimate

Valuation estimate

Number of profiles

Funding overview

Funding rounds

Investors

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M&A

Investments

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Frequently asked questions about Hope for PDCD Foundation