International Rett Syndrome Foundation

What International Rett Syndrome Foundation does

The International Rett Syndrome Foundation (IRSF) is a 501(c)(3) nonprofit foundation headquartered in Cincinnati, Ohio, that operates as the leading patient-focused organization dedicated exclusively to Rett syndrome. Founded in 1983 as the first nonprofit to focus on Rett and formed in its current shape in 2007 through the merger of the Rett Syndrome Research Foundation and the International Rett Syndrome Association, IRSF serves three constituencies: families and caregivers of individuals with Rett syndrome, academic and industry researchers studying MECP2 biology and gene-targeted therapeutics, and healthcare providers operating within its 18-clinic U.S. Center of Excellence Network and a 14-country international clinic footprint.

IRSF's product portfolio centers on a research and family-support platform rather than a single commercial offering. Core technology assets include the world's largest Rett Syndrome Natural History Study database (15+ years of data, 700+ patients informing Growth Charts), the IRSF Rett Syndrome Registry (longitudinal observational study on ClinicalTrials.gov NCT05432349), the My Rett Ally care-management web app (built on the mejo platform), and a Rett Syndrome Clinical Trial Finder that ingests listings from ClinicalTrials.gov. Programmatic products include the IRSF Rett Syndrome Scientific Meeting and ASCEND National Summit (500+ attendees, 50+ sessions), RettEd educational programs and webinars, Rett Forward Clinical Trial Master Class, the Comprehensive Care Guidelines and Resource Library, and the PRISM Study (a 2026 longitudinal presymptomatic biomarker study at UNC).

The revenue model is multi-stream nonprofit funding: individual donations (one-time, recurring, appreciated securities, planned gifts), corporate and foundation grants, signature fundraising events (Strollathons have raised nearly $17M since 2004; ASCEND 2026 sponsorships from Acadia, Neurogene, Taysha, UCB, and Anovo), cause-marketing promotions, and government/institutional support. The foundation reports that more than 80 cents of every donated dollar supports the mission, holds a Candid Platinum Seal of Transparency 2025, and has invested more than $60M cumulatively in research — including contributions to the FDA approval of trofinetide (DAYBUE), the first-ever approved Rett treatment, in March 2023.

Name

International Rett Syndrome Foundation

Legal name

International Rett Syndrome Foundation

Website

https://rettsyndrome.org

Company type

Private

Founded year

1983

Operating status

Operating

Headcount range

11–50 employees

Short description

The International Rett Syndrome Foundation (IRSF) is a 501(c)(3) nonprofit foundation that funds Rett syndrome research, operates the largest Rett Natural History Study database and Registry, and provides family support and advocacy services to caregivers, clinicians, and researchers across an 18-clinic U.S. Center of Excellence network and 14-country international clinic footprint.

Ownership category

akta.pro rank

Headquarters

HQ city

Cincinnati

HQ country

United States

HQ region

North America

Offices1 record

Markets served

GTM type

B2B and B2C

Offering type

Services

Cost components

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

Revenue model

1. Individual Donations: Tax-deductible donations from individuals via IRSF website. Designated funds include the Research to Reality Fund, Steven G. Kaminsky Scout Program, and Barrett Otis Research Fund for Males. Donations can be made as one-time or recurring gifts, appreciated securities (DTC/wire transfer), or planned/estate gifts. On average, more than 80 cents of every dollar donated supports the mission.
2. Fundraising Events: Strollathons (nationwide fundraiser that has raised nearly $17M and counting), golf tournaments (Rip It For Rett, Goals fore Gracie, Westbury Open), Raise a Glass for Rett, Rett Adventure, and other community-driven fundraisers.
3. Event Registration & Sponsorship: ASCEND National Summit registration fees (Rett Family Ticket, Social Events Only, Individual, Industry Member, Virtual tiers) and corporate sponsorships (Presenting, Diamond, Gold, Bronze). Scientific Meeting registration fees for researchers/clinicians.
4. Corporate & Foundation Grants: Corporate and foundation grants, partnership donations (e.g., $500,000 from Vezbi Super App in 2023), and annual support from foundations including Ella Foard Foundation, Borracho Pescador, Casting 4A Cure, Raising a Hand, and Johnson HVAC Distribution.
5. Promotions That Give Back: Cause-marketing partnerships where a percentage of sales benefit IRSF (e.g., Billy Footwear: 10% off + 5% to IRSF). Vehicle donations via CARS partnership.

Pricing tiers

Go-to-market motion1 record

Distribution channels8 records

Marketing channels10 records

Core offering

IRSF is a 501(c)(3) nonprofit foundation dedicated exclusively to Rett syndrome. It funds transformative research, stewards the world's largest Rett Syndrome Natural History Study database and Registry, provides family education and care-coordination resources (including the My Rett Ally web app), runs the IRSF Center of Excellence Network of clinical and research centers, and advocates for federal research funding and rare disease policy. Its programs serve families, researchers, clinicians, and industry partners across more than 14 countries.

Product overview

The International Rett Syndrome Foundation (IRSF) operates as a nonprofit foundation delivering a multi-program platform of research, family support, and advocacy services rather than a single unified product. Its core digital offerings include My Rett Ally (a care-management web app built on the mejo platform), the IRSF Rett Syndrome Registry (a longitudinal observational study feeding the largest Rett Natural History database in the world), and the Rett Syndrome Clinical Trial Finder (which ingests listings from ClinicalTrials.gov). Educational products include Rett Forward: A Clinical Trial Master Class, the RettEd Programs and RettEd Video Library, and the Communication Guidelines / The Hive professional network. Clinical resources include the Comprehensive Care Guidelines, the Transition of Care Toolkit, the IEP Checklist, Growth Charts, and curated CME Opportunities. Flagship live programs include the annual ASCEND National Summit (June 30 - July 3, 2026 in Prior Lake, MN) and the IRSF Rett Syndrome Scientific Meeting (June 29 - July 1, 2026). The foundation also stewards the Center of Excellence Network (15+ U.S. clinics), the PRISM Study, the Find a Clinic directory, Family Listening Sessions, and the Rett Treatment Pipeline information resource. Underpinning all of this is the IRSF Research Grants Program (Research to Reality Fund, Steven G. Kaminsky Scout Program, Barrett Otis Research Fund for Males), with over $60M invested since 1983 in breakthrough discoveries that helped identify MECP2 as the causal gene, supported the FDA approval of trofinetide (DAYBUE), and accelerated gene therapy programs (Taysha TSHA-102, Neurogene NGN-401).

Differentiator

Problem solved

Functional benefit

Brands

• My Rett Ally: Free web application (powered by mejo) specifically designed for Rett syndrome patients and families to simplify and enhance care coordination, including a medical journal, customizable tracking categories, and document storage.

Products and services

• My Rett Ally Free web application powered by mejo, designed exclusively for Rett syndrome patients and families. Helps caregivers simplify, organize, and share their child's medical and care information in one place, with an organized medical journal, customizable tracking categories, and document storage.
• IRSF Rett Syndrome Registry

Quantifiable outcome

• More than $60 million invested in Rett research breakthroughs over 40+ years
• +7 more outcomes

Named customers10 records

Segments5 records

Ideal customer profiles4 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Integration9 records

Feature7 records

Partnerships

28 partnerships are on record, tiered core, flagship and minor.

• Vezbi Super AppcoreTechnology or Integration · 3 January 2023Vezbi donated $500,000 to IRSF in January 2023 and is creating custom Micro-apps and Communities for use by IRSF and the community of families impacted by Rett syndrome that the foundation serves.
• Hive NetworkscoreTechnology or Integration · 18 October 2021Hive Networks announced a multi-year partnership with IRSF (Oct 2021) to provide a digital health platform to support the Rett community.
• Acadia PharmaceuticalsflagshipStrategic or Co-development PartnerAcadia Pharmaceuticals is the Presenting Sponsor of ASCEND 2026 Summit and Diamond sponsor across multiple years. Acadia developed and received FDA approval for trofinetide (DAYBUE) — the first-ever FDA-approved treatment for Rett syndrome (March 2023). IRSF played a key supporting role in enabling this milestone through its research funding and clinical trial infrastructure.
• NeurogeneflagshipStrategic or Co-development PartnerNeurogene is a Diamond Sponsor of ASCEND 2026. Conducting Phase 1/2 trial of NGN-401 investigational gene therapy for Rett syndrome. Neurogene has dosed pediatric patients in U.S. gene therapy clinical trial, with primary endpoints informed by the Rett Syndrome Natural History Study.
• Taysha Gene TherapiesflagshipStrategic or Co-development PartnerTaysha Gene Therapies is a Diamond Sponsor of ASCEND 2026. Running the REVEAL Phase 1/2/3 Pivotal Study of TSHA-102 gene therapy in females with Rett syndrome. Taysha's pivotal Part B trial design is informed by developmental milestones from IRSF's Rett Syndrome Natural History Study.
• UCBcoreGTM or Marketing PartnerUCB is a Gold Sponsor of ASCEND 2026 Rett Syndrome National Summit.
• AnovominorGTM or Marketing PartnerAnovo is a Bronze Sponsor of ASCEND 2026 Rett Syndrome National Summit.
• Midwest Rett Syndrome FoundationcoreStrategic or Co-development PartnerMidwest Rett Syndrome Foundation is a Bronze Sponsor of ASCEND 2026 and a partner organization. Board member Mitch Bleske (EVP & CFO of Bremer Financial) works with the Midwest Rett Syndrome Foundation.
• National Organization for Rare Disorders (NORD)coreStrategic or Co-development PartnerIRSF collaborates with NORD on rare disease advocacy, policy, and patient support. NORD's Rare Disease Educational Support Program provides financial assistance for registration costs and limited travel/lodging costs for ASCEND conference participation.
• Rare Diseases Clinical Research NetworkcoreStrategic or Co-development PartnerIRSF partners with the Rare Diseases Clinical Research Network (RDCRN) to advance clinical research in rare diseases including Rett syndrome.
• EveryLife Foundation for Rare DiseasescoreStrategic or Co-development PartnerIRSF collaborates with the EveryLife Foundation for Rare Diseases on policy advocacy and rare disease community initiatives.
• American Brain CoalitionminorStrategic or Co-development PartnerIRSF collaborates with the American Brain Coalition on brain-related disease advocacy initiatives.
• NIH (National Institutes of Health / NCATS)flagshipStrategic or Co-development PartnerIRSF collaborates with NIH, including NCATS. IRSF's CSO Dr. Dominique Pichard previously served as Director of the Division of Rare Diseases Research Innovation at NCATS. IRSF supports NIH initiatives on genetic medicines, newborn screening, and rare disease therapeutic development.
• Milken InstituteminorStrategic or Co-development PartnerIRSF partners with the Milken Institute on rare disease and biomedical research initiatives.
• Chan Zuckerberg InitiativeminorStrategic or Co-development PartnerIRSF partners with the Chan Zuckerberg Initiative on rare disease initiatives.
• Global GenescoreStrategic or Co-development PartnerIRSF collaborates with Global Genes on rare disease advocacy and community support.
• Child Neurology FoundationcoreStrategic or Co-development PartnerIRSF developed the Rett Syndrome Transition of Care Toolkit in collaboration with the Child Neurology Foundation as part of the ACP HVC pediatric to adult care transition project.
• MejocoreTechnology or IntegrationMejo powers the My Rett Ally web application — a free tool exclusively for the Rett community for care coordination. Mejo provides medical journal, tracking, and document storage features.
• Ella Foard FoundationminorStrategic or Co-development PartnerThe Ella Foard Foundation is one of IRSF's Supporters, providing generous annual donations to make IRSF's work possible.
• Borracho PescadorminorStrategic or Co-development PartnerBorracho Pescador is an IRSF Supporter providing generous annual donations.
• Casting 4A CureminorStrategic or Co-development PartnerCasting 4A Cure is an IRSF Supporter providing generous annual donations.
• Raising a HandminorStrategic or Co-development PartnerRaising a Hand is an IRSF Supporter. Photographer Dave Clements has documented musicians holding up their hands in support of Rett syndrome research; three bound collections of over 200 photos released.
• Johnson HVAC Distribution (Johnson Supply)minorStrategic or Co-development PartnerJohnson Supply (Executive VP is IRSF Board Chair Steve Wood) is an IRSF Supporter. Steve Wood and Johnson Supply support Raise a Glass and other IRSF fundraising initiatives.
• Billy FootwearminorGTM or Marketing PartnerPromotions That Give Back partnership — using a custom link, customers receive 10% off entire order and IRSF receives 5% toward its mission.
• CARS (Charitable Adult Rides & Services)minorGTM or Marketing PartnerVehicle donation partnership — IRSF works with CARS to convert donated vehicles into cash for the foundation. CARS arranges no-cost pickup within 24-72 business hours and provides tax documentation.
• The Assistance Fund (TAF)minorStrategic or Co-development PartnerThe Assistance Fund operates the Rett Syndrome Financial Assistance Program, providing financial assistance for FDA-approved treatments (including copays, deductibles, premiums, therapy administration costs, treatment-related travel, diagnostic and genetic testing).
• PAN FoundationminorStrategic or Co-development PartnerThe PAN Foundation provides underinsured patients with Rett syndrome access to disease-specific grants for out-of-pocket medication/treatment costs via the Rett syndrome fund.
• University of North Carolina Rett Syndrome CliniccoreStrategic or Co-development PartnerUNC Rett Syndrome Clinic (Yael Shiloh-Malawsky, MD / Diana Cejas, MD, MPH) is an IRSF Center of Excellence receiving research funding for the PRISM Study — a 2-year longitudinal presymptomatic biomarker study using non-invasive EEG assessments in infants/toddlers with pathogenic MECP2 variants.

Scale indicators15 records

Recent moves11 records

Direct peers

• Rett Syndrome Research Trust: A privately funded research-focused nonprofit dedicated exclusively to Rett syndrome. Directly comparable to IRSF in mission (research and family support) and disease focus, with overlapping scientific advisors and competing donor mindshare.
• CureDuchenne: Rare disease patient foundation focused on Duchenne muscular dystrophy research, family support, and industry partnerships. Highly comparable in operating model; new IRSF CEO Laura Hameed was previously its Executive Director.

Broad incumbents

• Parent Project Muscular Dystrophy (PPMD): Larger, established rare-disease patient foundation for Duchenne. Comparable in mission structure (research funding, Centers of Excellence, family education, gene therapy advocacy), but broader program footprint and longer gene therapy track record.
• Cystic Fibrosis Foundation: The archetypal rare-disease patient foundation; comparable in research-grant model, Center of Excellence network (analogous to IRSF's), industry co-funding approach, and ability to anchor late-stage therapeutic development (e.g., Trikafta/Kaftrio).
• National Organization for Rare Disorders (NORD): Umbrella rare-disease advocacy organization with which IRSF partners. Comparable in policy advocacy, member foundation services, and rare-disease educational programming (NORD's Rare Disease Educational Support Program supports ASCEND scholarships).

Emerging players

• National Fragile X Foundation: Single-disease patient foundation for Fragile X syndrome, a related neurodevelopmental disorder. Comparable in clinical research network model, family-centered education, and conference-based community building.
• Tuberous Sclerosis Alliance: Patient advocacy and research foundation for tuberous sclerosis complex. Comparable in clinic designation network, natural history and registry programs, and co-funding of mTOR-pathway drug development.
• Child Neurology Foundation: Foundation serving children with neurologic conditions, with which IRSF co-developed the Transition of Care Toolkit. Comparable in CME offerings, transition-of-care resources, and family-clinician partnership programs.

Regional players

• EveryLife Foundation for Rare Diseases: U.S.-focused rare disease policy and advocacy nonprofit; comparable in federal advocacy (Rare Disease Week), newborn screening priorities, and community grants. Complementary rather than competitive to IRSF.
• Global Genes: U.S.-based rare disease advocacy and education nonprofit. Comparable in rare-disease community programming, partnership network (IRSF is listed as a partner), and disease-awareness content production.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat6 records

Key risks6 records

Key highlights7 records

Customer concentration

• Xx.com/Rettsyndrome
• YouTubeyoutube.com/playlist?list=PLLkvctrhQY0P8dxoFW0zt0D7o5fENS43x
• Facebookfacebook.com/RettSyndrome
• LinkedInlinkedin.com/company/rettsyndrome.org

Revenue estimate

Valuation estimate

Number of profiles

Profiles10 records

Funding overview

Funding rounds

Investors

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M&A

Investments1 record

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Frequently asked questions about International Rett Syndrome Foundation