Chelsea’s Hope

What Chelsea’s Hope does

Chelsea's Hope Lafora Children Research Fund is a 501(c)(3) non-profit patient advocacy organization founded in 2007 and headquartered in Lexington, Kentucky. Named in memory of Chelsea Gerber (1990-2016), who was diagnosed with Lafora disease at age 15, the organization is the first and leading global advocacy body for Lafora disease — a rare, fatal glycogen storage disorder affecting an estimated 200 people worldwide. Its core activities center on funding and coordinating Lafora disease research, including the ION283 Safety Study at UT Southwestern Medical Center (FDA-approved August 2024, enrolling 10 patients), maintaining the Lafora Clinical Network Registry (launched July 2026), the Lafora Mutations Database, the Lafora Canine Registry, and the Madrid-based Lafora Disease Clinical Registry. Chelsea's Hope also operates family support programs (newly diagnosed guidance, bereavement support, G-tube care resources), educational content, and convenes annual international science symposiums and research roundtables. The organization played a central role in securing ICD-10 code G40.C for Lafora disease (effective October 1, 2023).

The organization's core "products" are not commercial technology platforms but rather mission-driven research and community infrastructure: research databases, patient and clinician registries, educational resources, and clinical trial funding coordination. Its underlying technology footprint includes the Probably Genetic integration for no-cost genetic testing in the US, peer-to-peer fundraising infrastructure via Givebutter and Network for Good, and an active presence across Facebook, Instagram, X, LinkedIn, YouTube, and TikTok. Chelsea's Hope does not develop proprietary technology and operates on a model of curating and connecting distributed scientific and community assets.

Chelsea's Hope generates revenue entirely through tax-deductible donations (individual contributions via mail and online), peer-to-peer fundraising campaigns, and grants from foundations including the Chan Zuckerberg Initiative (Rare As One Network), Horizon Therapeutics (#RAREis Global Advocate Grant, 2022), and the RTW Foundation ($100K Community Grant, December 2025). Cumulative fundraising since inception totals $5.5M, with >$1.3M raised in coordination with international partner organizations (A.I.L.A., Lafora France, Tempo Zero, AEVEL, Cel-Luz) toward the $1.5M ION283 Safety Study budget. The organization earns no product or service revenue and is governed by a volunteer Board of Directors led by President Jenifer Merriam, with a small operational staff including Scientific and Executive Director Kit Donohue, PhD, Communications Director Christine Kelly, Grants Coordinator Kait Fedor, and Clinical Program Lead Souad Messahel, PhD.

Name

Chelsea’s Hope

Legal name

Chelsea's Hope Lafora Children Research Fund

Website

https://chelseashope.org

Company type

Private

Founded year

2007

Operating status

Operating

Headcount range

1–10 employees

Short description

Chelsea's Hope is a 501(c)(3) non-profit that funds Lafora disease research, coordinates the ION283 Safety Study, maintains patient and clinical registries, and connects affected families globally through support programs, education, and peer-to-peer fundraising.

Ownership category

akta.pro rank

Product category

Rare Disease Patient Advocacy

NAICS

Individual and Family Services (6241), Other Individual and Family Services (62419)

SIC

Services-Social Services (8300), Services-Health Services (8000)

akta.pro primary industry

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

akta.pro secondary industries

Rare Disease & Special Needs Support Organizations (BPAGACAM), Health Systems Strengthening & Global Health Technical Assistance (BPADAEAH)

Keywords

• Rare disease advocacy
• Lafora disease research
• Patient advocacy nonprofit
• Clinical trial funding
• Patient registry services

Headquarters

HQ city

Sacramento

HQ country

United States

HQ region

North America

Offices1 record

Markets served

GTM type

B2C

Offering type

Services

Cost components

Personnel, Others, Marketing or Sales, Operations

Revenue model

1. Donations and Grants: Chelsea's Hope raises funds entirely through donations and grants to support Lafora disease research. This includes individual tax-deductible donations (mail-in checks and online), peer-to-peer fundraising campaign contributions, corporate-matched gifts, and foundation grants (e.g., RTW Foundation $100k grant, Chan Zuckerberg Initiative Rare As One grant, Horizon Therapeutics #RAREis grant). The organization is an IRS 501(c)(3) non-profit (EIN: 27-1008382) and contributions are federally tax-deductible as well as in many U.S. states.

Pricing tiers

Go-to-market motion1 record

Distribution channels5 records

Marketing channels9 records

Core offering

Chelsea's Hope is a 501(c)(3) non-profit patient advocacy organization that raises funds for Lafora disease research, coordinates international patient registries and clinical networks, connects affected families globally, and advances therapeutic development through partnerships with biotech companies and academic medical centers. The organization maintains unique data assets including a mutations database and canine registry, supports the ION283 ASO therapy safety study at UT Southwestern, and serves as the primary hub for the global Lafora disease community of approximately 200 affected individuals worldwide.

Product overview

Chelsea's Hope operates as a patient advocacy nonprofit offering a portfolio of interconnected services centered on Lafora disease support, research acceleration, and family connection. The core offerings include the Lafora Clinical Network Registry (a clinician directory connecting patients to specialists), the ION283 Safety Study (actively recruiting patients for an ASO therapy clinical trial), the Lafora Mutations Database and Lafora Canine Registry (research databases for scientists), and comprehensive Lafora Disease Education Resources including a therapy pipeline tracker. Family support services encompass newly diagnosed guidance, symptom checkers via a partnership with Probably Genetic, bereavement support, and a private Facebook community. Research advancement is facilitated through annual international symposiums and research roundtables. The organization functions as a hub connecting patients, families, researchers, clinicians, and biotech partners to accelerate treatments for this rare neurodegenerative glycogen storage disease.

Differentiator

Problem solved

Functional benefit

Products and services

• Lafora Clinical Network Registry A centralized registry connecting patients and families to clinicians with experience treating Lafora disease, featuring clinician profiles with expertise, services, and contact information. Clinicians apply to be listed; families search the registry to find knowledgeable providers.
• ION283 Safety Study A 2-year human safety study at UT Southwestern Medical Center testing ION283 ASO therapy for Lafora disease. Enrolls 10 patients aged 10-18 with genetically confirmed diagnosis, with dose escalation from 15mg to 30mg. Chelsea's Hope fundraises to cover the estimated $1.5 million in clinical costs.
• Lafora Mutations Database A collaborative database of genetic mutations for Lafora disease researchers and scientists. Researchers can request access to view reported mutations, and novel mutations can be reported via a dedicated form.
• Lafora Canine Registry and Database A registry and database compiling genetic data from dogs affected by Lafora disease to advance research understanding of the disease and potential treatments in canine models.
• Lafora Disease Symptom Checker An interactive assessment tool developed in partnership with Probably Genetic that helps users determine eligibility for no-cost genetic testing for pediatric epilepsy disorders, including Lafora disease. Testing is available in the United States.
• Family Support Programs Support services for Lafora disease families including newly diagnosed guidance, tips for families, bereavement support, G-tube care tips, and connection to caregiver networks and monthly virtual meetups.
• Lafora Disease Registry (Madrid) A patient registry database for Lafora disease patients, operated in partnership with Madrid, to connect patients with research and prepare for future clinical trials.

Quantifiable outcome

• Raised $5.5 million for Lafora disease research since 2007
• +4 more outcomes

Named customers2 records

Segments3 records

Ideal customer profiles3 records

Technology focussed No

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Integration1 record

Partnerships

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

• Elpida TherapeuticscoreStrategic or Co-development Partner · 1 August 2026Elpida Therapeutics acquired ION283 from Ionis Pharmaceuticals and is advancing this potential therapy for Lafora disease. CEO Terry Pirovolakis shared a letter with the Lafora community introducing the company and its plans. Elpida is a nonprofit biotech company focused on advancing rare disease therapies.
• Fondazione TelethoncoreStrategic or Co-development Partner · 1 July 2025Fondazione Telethon is a non-profit pharmaceutical organization that develops and produces gene therapies. Noventia was in final stages of formalizing an agreement with Telethon for clinical development of ION283. Telethon reaffirmed its commitment to the Lafora community despite the asset transfer from Ionis. Telethon has a 35+ year track record of advancing rare genetic disease therapies and became the first charity in the world to have a gene therapy developed in its own laboratories authorized for the U.S. market.
• Ionis PharmaceuticalscoreStrategic or Co-development Partner · 4 September 2024Ionis Pharmaceuticals developed ION283, an antisense oligonucleotide (ASO) therapy targeting GYS1 to reduce glycogen synthase and prevent Lafora body formation. Ionis provided the drug free of charge for the safety study and held the IND application. In September 2024, Ionis licensed ION283 to Noventia Pharmaceuticals. Subsequently, Noventia transferred development rights to Elpida Therapeutics (August 2026).
• Noventia PharmaceuticalsmajorStrategic or Co-development Partner · 1 September 2024Noventia Pharmaceuticals licensed ION283 from Ionis Pharmaceuticals in September 2024. Noventia owns the license for ION283 and guaranteed drug supply for the 10 patients in the safety study. Development rights were subsequently transferred to Elpida Therapeutics.
• UT Southwestern Medical CentercoreStrategic or Co-development PartnerChelsea's Hope partners with UT Southwestern Medical Center in Dallas, Texas to conduct the ION283 Safety Study led by Dr. Berge Minassian (Chief of Pediatric Neurology). The 2-year study enrolls 10 Lafora disease patients who receive ASO therapy doses every 3 months. Chelsea's Hope fundraises to cover clinical costs while UT Southwestern provides clinical infrastructure, regulatory expertise, and FDA IND application management. The study also benefits from Ronald McDonald House partnerships for patient family accommodation.
• Probably GeneticmajorStrategic or Co-development PartnerChelsea's Hope partnered with Probably Genetic to offer a no-cost genetic testing program for pediatric epilepsy disorders, including Lafora disease. Families complete a symptom checker quiz online to determine eligibility. If eligible, a sample collection kit is mailed to the home with return pickup. Results are delivered in 6-8 weeks with optional genetic counseling. Testing is available only in the United States at this time.
• Chan Zuckerberg Initiative (CZI) — Rare As One NetworkmajorStrategic or Co-development PartnerChelsea's Hope is a member of the Rare As One Network, powered by the Chan Zuckerberg Initiative. The network supports rare disease patient advocacy organizations with resources, collaboration opportunities, and funding (including a patient-partnered collaborations grant in 2023). The partnership helps Chelsea's Hope connect with other rare disease organizations and access scientific and operational resources.
• National Organization for Rare Disorders (NORD)majorGTM or Marketing PartnerChelsea's Hope is a Platinum member of NORD for 2025. NORD offers resources for rare disease patients including financial assistance and guidance. NORD membership provides Chelsea's Hope with credibility, access to NORD's network of rare disease organizations, and visibility within the rare disease community.
• Rare Epilepsy Network (REN)minorGTM or Marketing PartnerChelsea's Hope is a proud member of the Rare Epilepsy Network (REN), connecting rare epilepsy advocacy organizations to share resources, research, and best practices for patient advocacy.
• A.I.L.A. (Associazione Italiana Lafora)majorStrategic or Co-development PartnerA.I.L.A. is the Italian Lafora disease patient organization. Chelsea's Hope partners with A.I.L.A. on international fundraising for the ION283 Safety Study, research collaboration, and awareness events. A.I.L.A. logo appears on Chelsea's Hope Partners section alongside other international Lafora organizations.
• Lafora FrancemajorStrategic or Co-development PartnerLafora France is the French Lafora disease patient organization. Together with Chelsea's Hope, A.I.L.A., TempoZero, Cel-Luz, and the Weiss family, Lafora France co-funded the ION283 Safety Study, raising more than $1.3 million collectively. Lafora France also maintains its own dedicated fundraising page for the safety study.
• Tempo ZeromajorStrategic or Co-development PartnerTempo Zero is an Italian Lafora disease patient organization (ODV). It is part of the international coalition co-funding the ION283 Safety Study alongside Chelsea's Hope, A.I.L.A., Lafora France, Cel-Luz, and the Weiss family.
• AEVEL (Asociación Española para Vencer la Enfermedad de Lafora)minorStrategic or Co-development PartnerAEVEL is the Spanish Lafora disease patient organization. Its logo appears on Chelsea's Hope Partners section alongside other international Lafora organizations.
• Cel-Luz (Asociación CEL LUZ)majorStrategic or Co-development PartnerCel-Luz is a Spanish Lafora disease patient organization. It is part of the international coalition co-funding the ION283 Safety Study alongside Chelsea's Hope, A.I.L.A., Lafora France, Tempo Zero, and the Weiss family.
• LibrareyminorGTM or Marketing PartnerLibrarey is a resource collection platform by rare disease families, for rare disease families. Chelsea's Hope highlights Librarey on its Resources page as a recommended tool for the Lafora community to find and share resources.
• Ronald McDonald HouseminorOthersRonald McDonald House provides accommodation and meals for patients and their families at UT Southwestern Medical Center, supporting families who travel to Dallas for the ION283 Safety Study treatment visits. Chelsea's Hope works with Ronald McDonald House to locate financial support resources for families needing assistance.

Scale indicators6 records

Recent moves6 records

Expansion highlights6 records

Broad incumbents

• Chan Zuckerberg Initiative — Rare As One Network: CZI's Rare As One Network is the funding and convening platform Chelsea's Hope is a member of. Supports dozens of rare disease advocacy organizations with grants and collaboration resources — broader in scope but a peer in mission-driven rare disease patient advocacy.
• National Organization for Rare Disorders (NORD): NORD is the umbrella US organization for rare disease patient advocacy, of which Chelsea's Hope is a Platinum member for 2025. Operates broadly across 7,000+ orphan diseases rather than specializing in Lafora, but provides policy, financial assistance, and network access that Chelsea's Hope leverages.

Direct peers

• Asociación CEL LUZ: Cel-Luz is a Spanish Lafora disease patient organization that co-funds the ION283 Safety Study with Chelsea's Hope. Identical disease-focus, patient-advocacy mission, and cross-border fundraising coalition structure.
• A.I.L.A. (Associazione Italiana Lafora): A.I.L.A. is the Italian Lafora disease patient organization and a direct coalition partner of Chelsea's Hope, co-funding the ION283 Safety Study alongside it. Operates the same disease-specific advocacy, family support, and fundraising model for Italian families.
• Tempo Zero: Tempo Zero is an Italian Lafora disease patient organization (ODV) and co-funder of the ION283 Safety Study alongside Chelsea's Hope. Operates an essentially identical patient advocacy and research fundraising model for the same ultra-rare disease population.
• AEVEL (Asociación Española para Vencer la Enfermedad de Lafora): AEVEL is the Spanish Lafora disease patient organization, displayed as a partner on Chelsea's Hope's site. Direct disease-specific peer with comparable mission, governance, and donor base structure for Spanish families.
• Lafora France: Lafora France is the French Lafora disease patient organization, part of the international coalition co-funding the ION283 Safety Study with Chelsea's Hope. Same disease focus, same advocacy and fundraising model, different national geography.

Emerging players

• Parent Project Muscular Dystrophy (PPMD): PPMD is a parent-driven nonprofit advancing Duchenne muscular dystrophy research, registries, and clinical trials. Closely analogous to Chelsea's Hope's structure: family-led, focused on a single rare pediatric disease, coordinating biotech partnerships and FDA-approved trials.
• Cure SMA: Cure SMA is a US-based rare disease advocacy organization for spinal muscular atrophy. Operates a comparable model — patient registries, biotech-funded trial advancement, multi-million-dollar annual fundraising, and FDA-approved therapy launches — for a similarly devastating pediatric genetic disease.

Regional players

• Rare Epilepsy Network (REN): REN connects rare epilepsy advocacy organizations, including Chelsea's Hope. Operates a similar advocacy and family-support mission but at the broader rare-epilepsy category level rather than for a single ultra-rare disease.

Market position

Strengths5 records

Competitive moat5 records

Key risks6 records

Key highlights7 records

Customer concentration

• Xx.com/Chelseashopela1
• YouTubeyoutube.com/@chelseashopelaforacure
• Facebookfacebook.com/chelseashopelaforacure
• LinkedInlinkedin.com/company/chelsea-s-hope-lafora-children-research-fund-usa
• Instagraminstagram.com/chelseashopelaforacure
• TikToktiktok.com/@chelseashopelaforacure

Compliance4 records

Revenue estimate

Valuation estimate

Number of profiles

Profiles11 records

Funding overview

Funding rounds

Investors

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M&A

Investments

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Frequently asked questions about Chelsea’s Hope