Parent Project Muscular Dystrophy

Name

Parent Project Muscular Dystrophy

Legal name

The Parent Project for Muscular Dystrophy Research Inc

Website

https://parentprojectmd.org

Company type

Private

Founded year

1994

Operating status

Operating

Headcount range

11–50 employees

Ownership category

akta.pro rank

Headquarters

HQ city

Washington

HQ country

United States

HQ region

North America

Offices1 record

Markets served

GTM type

B2B and B2C

Offering type

Services

Cost components

Personnel, Operations, Marketing or Sales, Others, Infrastructure

Revenue model

1. Donations and Fundraising: PPMD generates revenue through donations from individuals, corporate sponsors, and fundraising events including the Race to End Duchenne program (raised over $19 million since 2005), Sam's Night event (raised over $10 million since inception), and DIY Fundraising initiatives.
2. Venture Philanthropy Grants: PPMD provides funding to biotech companies through its Venture Pathways program, which functions as venture philanthropy to accelerate therapeutic development for Duchenne. Examples include $250,000 to Secretome Therapeutics and $400,000 to MyoGene Bio.

Go-to-market motion1 record

Distribution channels3 records

Marketing channels8 records

Core offering

PPMD is a nonprofit patient advocacy organization that accelerates research, care, and policy to end Duchenne and Becker muscular dystrophy. It runs The Duchenne Registry (a patient-powered data network), funds biotech companies through the Venture Pathways venture-philanthropy program, certifies care centers, provides free genetic testing via Decode Duchenne, publishes care guidelines, and hosts the world's largest Duchenne annual conference alongside advocacy and community support programs for affected families.

Product overview

Parent Project Muscular Dystrophy operates as a nonprofit patient advocacy organization rather than a commercial product company. Its core offering is The Duchenne Registry, a patient-powered data network that collects longitudinal information from individuals with Duchenne and Becker muscular dystrophy. Supporting programs include the Venture Pathways venture-philanthropy initiative, the Certified Duchenne Care Center Program ensuring standardized comprehensive care, the interactive Duchenne Drug Development Pipeline documenting therapies in development, and the Decode Duchenne free genetic testing program. Additional resources include Duchenne Care Guidelines, emergency care protocols, educational webinars, the Living Duchenne Podcast, and community connection programs. The organization's fundraising efforts include the Race to End Duchenne endurance program and annual events. PPMD's impact includes over $55 million invested in research, 10 years of average lifespan extension attributed to care advances, and $850 million in leveraged federal funding.

Differentiator

Problem solved

Functional benefit

Products and services

• The Duchenne Registry A patient-powered data network collecting longitudinal information from individuals with Duchenne and Becker muscular dystrophy and female carriers. The registry has operated for over 10 years, with anonymous data shared with researchers to speed therapy development, support clinical trial recruitment, and improve understanding of disease progression.
• PPMD Venture Pathways A venture-philanthropy funding initiative that provides capital to biotech companies developing Duchenne and Becker muscular dystrophy therapies, including gene editing platforms and cardiac cell therapies. Examples include $250,000 to Secretome Therapeutics for STM-01 cardiac cell therapy and $400,000 to MyoGene Bio for a gene editing therapy.
• Certified Duchenne Care Center Program An accreditation program that ensures hospitals and clinics provide comprehensive Duchenne care in agreement with CDC Care Considerations. Hospitals apply to be designated as Certified Duchenne Care Centers, strengthening the national network of specialized Duchenne care providers.
• Decode Duchenne Free Genetic Testing Program A free genetic testing and counseling program for individuals with Duchenne and Becker muscular dystrophy, designed to identify genetic variants and inform treatment decisions.
• Duchenne Care Guidelines Comprehensive care guidelines covering cardiac, pulmonary, bone/joint, emergency, and other areas of Duchenne management, organized by area of care and stage of disease progression. Includes emergency care and surgery/anesthesia precautions for clinicians and families.
• Duchenne Drug Development Pipeline An interactive pipeline documenting potential Duchenne treatments in development, including therapeutic approaches that restore or replace dystrophin and those that treat symptoms such as muscle protection and fibrosis reduction.
• Race to End Duchenne PPMD's signature endurance fundraising program involving marathons and races that has raised more than $19 million since 2005 to support Duchenne research, care, and advocacy.
• Sam's Night Fundraising Event An annual fundraising gala for PPMD, first held in 2017 and celebrating its 20th anniversary in 2025, having raised more than $10 million since inception.
• Annual Duchenne Conference The world's largest gathering for the Duchenne and Becker community, with the 32nd iteration scheduled for June 2026 in Orlando, Florida, expecting more than 1,000 attendees including families, researchers, clinicians, and industry partners.
• PPMD Advocacy Conference An annual advocacy event bringing 120+ Duchenne advocates to Capitol Hill to promote federal policies supporting Duchenne research, care, and advocacy.
• Living Duchenne Podcast A podcast series providing educational content and community stories for families affected by Duchenne muscular dystrophy.

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

Four partnerships are on record, tiered core.

• Mesoblast LimitedcoreGTM or Marketing Partner · 8 April 2026Mesoblast is collaborating with PPMD to support patient identification and trial awareness for its FDA-cleared registrational clinical trial evaluating Ryoncil in pediatric DMD patients. PPMD helps connect eligible patients with the trial.
• Cooperative International Neuromuscular Research Group (CINRG)coreStrategic or Co-development Partner · 6 March 2026PPMD partnered with CINRG to advance the expanded Duchenne Natural History Study (eDNHS), gathering long-term data on disease progression to facilitate therapy development and improve clinical practices. The study is active and enrolling at ten sites worldwide across North America, Australia, and India.
• University of California, DaviscoreStrategic or Co-development Partner · 6 March 2026UC Davis is the sponsor of the expanded Duchenne Natural History Study (eDNHS) and serves as a principal investigation site for this global long-term observational study.
• Broad InstitutecoreStrategic or Co-development Partner · 14 November 2024PPMD organized a symposium at the Broad Institute bringing together researchers, companies, and FDA officials to address challenges in conducting AAV gene therapy clinical trials in seropositive patients with pre-existing antibodies.

Scale indicators10 records

Recent moves6 records

Expansion highlights5 records

Direct peers

• Muscular Dystrophy Association: The largest U.S. muscular dystrophy nonprofit, covering Duchenne, Becker, and many other neuromuscular diseases. Directly comparable to PPMD as a patient advocacy, research funding, and care guidelines organization, though broader in disease scope and historically larger in fundraising scale.
• Cure Duchenne: A disease-specific nonprofit focused exclusively on finding a cure for Duchenne muscular dystrophy. Closely comparable to PPMD in mission, venture philanthropy funding of biotechs, and patient registry operations, though smaller in scale.
• Jett Foundation: A Duchenne muscular dystrophy-focused nonprofit providing patient support, fundraising, and research funding. Comparable to PPMD in disease focus and patient-family services, though with a smaller national footprint.
• Charley's Fund: A venture philanthropy nonprofit focused on funding Duchenne muscular dystrophy research and therapeutic development. Comparable to PPMD's Venture Pathways in approach and mission, though smaller in scale and assets.

Broad incumbents

• Cystic Fibrosis Foundation: The archetypal disease-focused venture philanthropy nonprofit, credited with funding development of transformative CF therapies. Comparable to PPMD as a model for patient advocacy organizations funding biotech R&D and driving care standards, though operating in a different (and now better-funded) disease area.
• National Organization for Rare Disorders (NORD): A U.S. umbrella organization serving as the collective voice for the rare disease community on policy, research funding, and patient access. Comparable to PPMD on the policy advocacy dimension, though NORD's multi-disease scope makes it more of an ecosystem partner than a direct competitor.

Regional players

• Action Duchenne: A UK-based Duchenne muscular dystrophy charity providing patient advocacy, research funding, and community support. Comparable to PPMD in mission and program mix, but operating primarily in the United Kingdom and not directly competing in U.S. territory.

Others

• Parent to Parent USA: A national nonprofit connecting parents of children with disabilities for peer support. Comparable to PPMD's community support and Connect Groups model, though not disease-specific and focused on peer matching rather than research funding.
• EveryLife Foundation for Rare Diseases: A rare disease policy advocacy organization focused on accelerating biotech innovation and patient access. Comparable to PPMD on federal advocacy (FDA, NIH, Congressional engagement), though focused broadly across rare diseases.
• Parent Heart Watch: A national nonprofit focused on preventing sudden cardiac arrest in youth through awareness, screening, and advocacy. Comparable to PPMD in the parent-led patient advocacy nonprofit model and focus on cardiac complications of genetic disease (cardiomyopathy is the leading cause of death in Duchenne).

Market position

Strengths5 records

Weaknesses5 records

Competitive moat5 records

Key risks5 records

Key highlights7 records

Customer concentration

• Xx.com/parentprojectmd
• YouTubeyoutube.com/user/ParentProjectMD
• Facebookfacebook.com/parentprojectmd
• LinkedInlinkedin.com/company/parent-project-muscular-dystrophy
• Instagraminstagram.com/parentprojectmd

Revenue estimate

Valuation estimate

Number of profiles

Profiles2 records

Subsidiaries2 records

Funding overview

Funding rounds

Investors

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M&A

Investments12 records

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Frequently asked questions about Parent Project Muscular Dystrophy