M L D FOUNDATION

What M L D FOUNDATION does

MLD Foundation is a 501(c)(3) nonprofit patient advocacy organization founded in 2001 and headquartered in West Linn, Oregon, that operates exclusively in the metachromatic leukodystrophy (MLD) disease space. Its mission is organized around a four-part C.A.R.E. framework (Compassion, Awareness, Research, Education), and it serves three primary constituencies: MLD-affected families (350+ active families across more than 15 countries), clinicians and researchers studying the disease, and pharmaceutical developers of MLD therapies.

The foundation's core programmatic offerings include a moderated Family Discussion List operating since 2001, a Family Facebook Group, an annual Family Conference, a Patient Powered Registry aggregating clinical data across 300+ ARSA mutations, Newborn Screening Advocacy, and a formalized Gene Therapy Access Support service. Its principal technology asset is the MLD Genomic Information & Research Platform, an interactive predictive model built on ARSA activity data spanning 300+ mutations and published genotype/phenotype correlations, currently under publishing embargo, intended for clinicians, families, and therapeutic decision-making.

The business model is donation-based: contributions are tax-deductible under 501(c)(3) status, supplemented by corporate sponsorship from pharmaceutical partners (Orchard Therapeutics, Takeda, Sanofi) and academic collaborators (Broad Institute/Mass General Brigham, SR-Tiget). Revenue supports community programs, an annual conference, advocacy, and research funding rather than commercial product sales. Recent strategic milestones include FDA approval of Lenmeldy gene therapy on March 18, 2024, RUSP nomination in July 2024, addition of MLD to the federal Recommended Uniform Screening Panel on December 16, 2025, and New York becoming the first U.S. state to begin MLD newborn screening on September 12, 2025.

Name

M L D FOUNDATION

Legal name

MLD Foundation

Website

https://mld.foundation

Company type

Private

Founded year

2001

Operating status

Operating

Short description

MLD Foundation is a 501(c)(3) nonprofit patient advocacy organization supporting families affected by metachromatic leukodystrophy through community programs, a patient registry, a genomic research platform, and federal/state newborn screening advocacy, serving 350+ active families across more than 15 countries.

Ownership category

akta.pro rank

Product category

Rare Disease Patient Advocacy

NAICS

Voluntary Health Organizations (813212), Grantmaking Foundations (813211), Other Individual and Family Services (624190)

SIC

Services-Social Services (8300), Services-Commercial Physical & Biological Research (8731), Services-Misc Health & Allied Services, Nec (8090)

akta.pro primary industry

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

akta.pro secondary industries

Health & Medical Research Grantmaking Foundations (BPAGAKAL), Neurogenetics & Rare Neurologic Diseases (HLAKAIAO)

Keywords

• Rare disease advocacy
• Patient support services
• Newborn screening advocacy
• Genetic disease research
• Family community support

Headquarters

HQ city

West Linn

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, Technology or R&D, Others

Revenue model

1. Donations: MLD Foundation is a 501(c)(3) nonprofit organization that raises funds through donations from individuals, families, and supporters. Donations are tax-deductible and used to further activities in the foundation's four C.A.R.E. areas of focus: Compassion, Awareness, Research, and Education.

Pricing tiers

Go-to-market motion1 record

Distribution channels2 records

Marketing channels9 records

Core offering

MLD Foundation is a 501(c)(3) nonprofit organization that provides support, advocacy, education, and research programs for families affected by Metachromatic Leukodystrophy (MLD), an ultra-rare genetic neurometabolic disease. The foundation operates across four C.A.R.E. mission areas — Compassion, Awareness, Research, and Education — delivering direct family support, newborn screening policy advocacy, MLD-specific genomic resources for clinicians, and annual family conferences connecting patients worldwide.

Product overview

MLD Foundation serves as the premier family resource for Metachromatic Leukodystrophy information and support since 1999, operating a comprehensive website portal that integrates multiple support services including a genomic research platform for severity prediction, patient-powered registry, newborn screening advocacy program, and annual family conferences. The organization provides education about MLD disease, therapies including gene therapy (Lenmeldy) and enzyme replacement therapy, peer support through discussion lists and Facebook groups, and advocacy for newborn screening implementation across U.S. states and federal RUSP. MLD Foundation's C.A.R.E. mission encompasses Compassion, Awareness, Research, and Education, focusing exclusively on MLD support and advancement.

Differentiator

Problem solved

Functional benefit

Products and services

• MLD Family Conference An annual in-person conference (held in North America and occasionally internationally) that brings together MLD families, doctors, researchers, and caregivers for multi-day education, peer support, clinical updates, and community connection. Includes the trademarked MLD Butterfly Ceremony and MLD Newborn Screening Academy educational track. Registration is heavily subsidized through grants and donations.
• MLD Patient-Powered Registry

Quantifiable outcome

• MLD added to the RUSP (Recommended Uniform Screening Panel) on December 16, 2025, enabling identification of MLD babies at birth across the US
• +3 more outcomes

Named customers1 record

Segments3 records

Ideal customer profiles3 records

Technology focussed No

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature1 record

Partnerships

Eleven partnerships are on record, tiered core, moderate and minor.

• Takeda Pharmaceutical CompanycoreStrategic or Co-development Partner · 1 January 2019Takeda (which acquired Shire in early 2019) is developing SHP-611, an enzyme replacement therapy for MLD, through the EMBOLDEN Phase II clinical trial. MLD Foundation has been working with Shire (now Takeda) since 2007 on this therapy. Takeda sponsored the 2024 MLD Family Conference as a Gold sponsor.
• Orchard TherapeuticscoreGTM or Marketing Partner · 1 January 2017Orchard Therapeutics acquired rights to commercialize the San Raffaele Telethon Institute's OTL-200 gene therapy (marketed as Lenmeldy in the US and Libmeldy in Europe). MLD Foundation works alongside Orchard to support patient access to gene therapy, provides physician and family navigation support, and participates in the Orchard Assist patient support program. Orchard sponsored the 2024 MLD Family Conference. MLD Foundation has been working with Orchard since 2017.
• San Raffaele Telethon Institute for Gene Therapy (SR-Tiget)coreStrategic or Co-development Partner · 1 January 2010SR-Tiget in Milan, Italy developed the ex-vivo lentiviral gene therapy OTL-200. MLD Foundation has been working with the Milano researchers and clinicians for over two decades (since 1995). The first-in-human clinical trial of OTL-200 started in April 2010 at this institute. The foundation continues to support patient navigation to this therapy.
• Children's Hospital of Philadelphia (CHOP)coreStrategic or Co-development PartnerMLD Foundation's Medical & Scientific Advisory Board includes CHOP-affiliated neurologists Dr. Laura Adang and Dr. Adeline Vanderver (who leads the CHOP Leukodystrophy Center). CHOP's Program for Neurodevelopment in Rare Disorders (NDRD) is referenced as a resource.
• University of MinnesotacoreStrategic or Co-development PartnerDr. William Krivit at University of Minnesota first inspired the Suhr family to begin serving the MLD community. The university is working to open a US Pre-Approval/Compassionate Access site for Lenmeldy gene therapy and is likely to be among the first to provide commercial access to US patients.
• Everylife FoundationmoderateStrategic or Co-development PartnerEverylife Foundation hosted an external Expert Evidence Review of all evidence supporting RUSP addition of MLD on September 12, 2025. MLD Foundation participates in EveryLife Community Congress calls and collaborates on rare disease policy initiatives.
• Ryan HouseminorStrategic or Co-development PartnerArizona-based organization providing respite and palliative care for children. Christine Hoffmann, MLD Foundation board member, is involved with Ryan House, representing a community care partnership.
• GlaxoSmithKline (GSK)minorStrategic or Co-development PartnerGSK acquired intellectual property from the San Raffaele Telethon Institute in October 2010 before Orchard Therapeutics acquired the IP from GSK in April 2018. This represents a historical development partnership in the gene therapy's path to commercialization.
• SanofiminorGTM or Marketing PartnerSanofi is listed as a Silver sponsor of the 2024 MLD Family Conference, supporting the foundation's educational and community programs.
• Foundazione Telethon (Just Like Home Program)moderateStrategic or Co-development PartnerFoundazione Telethon's Just Like Home program helps families relocating to Italy for gene therapy with practical, psychological, language, and cultural support, and provides financial support applications. MLD Foundation refers families to this program.
• MLD Doctors and Clinical Experts NetworkcoreStrategic or Co-development PartnerMLD Foundation maintains a network of MLD medical experts including specialists at Mayo Clinic (Dr. Marc Patterson), University of Padua (Dr. Alessandra Biffi), Cambridge University (Prof. Timothy Cox), Copenhagen (Dr. Christine I. Dali), UPMC Children's Hospital, and others for family referrals and clinical guidance.

Scale indicators4 records

Recent moves8 records

Expansion highlights6 records

Broad incumbents

• National Organization for Rare Disorders (NORD): Largest US rare disease umbrella advocacy organization representing ~300 disease-specific member organizations. Comparable in policy advocacy, patient support programs, and federal RUSP engagement, but operates broadly across conditions rather than focusing on a single disease.
• Global Genes: Global rare disease advocacy organization providing toolkits, education, and community-building resources to rare disease foundations. MLD Foundation operates in the same ecosystem but at the disease-specific layer, while Global Genes serves as an enabling umbrella.
• EveryLife Foundation for Rare Diseases: Public policy advocacy organization for rare disease community that hosted the external Expert Evidence Review supporting MLD's RUSP nomination. Operates the Community Congress and Rare Disease Legislative Advocates programs; overlapping policy-advocacy mission but broader across all rare diseases.

Direct peers

• Myelin Project: Nonprofit focused on myelin disorders including multiple leukodystrophies, funding research and supporting families. Directly comparable mission overlap in leukodystrophies, though broader in scope across multiple myelin diseases rather than exclusively MLD.
• Parent Project Muscular Dystrophy (PPMD): Disease-specific advocacy nonprofit for Duchenne muscular dystrophy, leading newborn screening and therapy access advocacy. Comparable in mission (rare genetic neuromuscular disease), advocacy playbook (federal and state policy), and pharma partnerships (Sarepta, Pfizer).
• Hunter's Hope Foundation: Family-founded nonprofit focused on Krabbe disease and other leukodystrophies, providing newborn screening advocacy, family support, and research funding. Most directly comparable leukodystrophy patient advocacy organization with parallel mission, RUSP policy work, and pharma partnership model.
• Cure SMA: Disease-specific nonprofit advocating for spinal muscular atrophy patients, families, and research. Highly comparable operating model — family-founded, drives newborn screening policy, partners with pharma (Biogen, Novartis, Roche) on gene therapy access, and runs annual family conferences.

Regional players

• CHOP Leukodystrophy Center of Excellence: Hospital-affiliated clinical and research program (Children's Hospital of Philadelphia, led by Dr. Adeline Vanderver) focused on leukodystrophies including MLD. Comparable in scientific mission and patient referral relationship, but operates as clinical provider rather than advocacy nonprofit.

Others

• Orchard Therapeutics: Commercial developer of Lenmeldy/Libmeldy gene therapy for MLD, with MLD Foundation serving as patient navigation and engagement partner. Closest commercial counterparty and the entity whose commercial success most directly validates the foundation's mission impact.
• Aldevron Foundation / Aldevron (now Danaher): Plasmid and protein manufacturer supporting gene therapy developers including Orchard Therapeutics. Adjacent ecosystem participant whose commercial products underpin the gene therapies MLD Foundation helps families access.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat6 records

Key risks6 records

Key highlights7 records

Customer concentration

• Facebookfacebook.com/MLDfoundation
• LinkedInlinkedin.com/company/mldfoundation

Revenue estimate

Valuation estimate

Number of profiles

Profiles5 records

Funding overview

Funding rounds

Investors

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M&A

Investments

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Frequently asked questions about M L D FOUNDATION