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| Parent Project Muscular Dystrophy | |
|---|---|
| Name | Parent Project Muscular Dystrophy |
| Founded | 1991 |
| Founder | Celeste and Jeff Messing |
| Location | New York, United States |
| Focus | Duchenne muscular dystrophy, Becker muscular dystrophy, neuromuscular disease advocacy |
| Key people | Pat Furlong |
Parent Project Muscular Dystrophy
Parent Project Muscular Dystrophy is a United States-based nonprofit advocacy organization focused on Duchenne muscular dystrophy and Becker muscular dystrophy. Founded by families affected by neuromuscular disease, the organization engages with federal agencies, pharmaceutical companies, academic centers, and international partners to accelerate treatments. Its activities intersect with major research institutions, patient advocacy groups, regulatory agencies, and private foundations.
The organization was established in 1991 by families influenced by activism from disability rights advocates and grassroots movements such as those associated with March of Dimes, Spinal Muscular Atrophy Foundation, American Heart Association, Cystic Fibrosis Foundation, Susan G. Komen for the Cure, Juvenile Diabetes Research Foundation, ALS Association, Alzheimer's Association, Parkinson's Foundation, March for Babies, Make-A-Wish Foundation, St. Jude Children's Research Hospital, Gates Foundation, Robert Wood Johnson Foundation, Ford Foundation, MacArthur Foundation, Carnegie Corporation, Rockefeller Foundation, W.K. Kellogg Foundation, Kresge Foundation, Open Society Foundations, Howard Hughes Medical Institute, Howard University Hospital, Johns Hopkins Hospital, Mayo Clinic, Cleveland Clinic, Massachusetts General Hospital, Mount Sinai Health System, Columbia University, Harvard University, Stanford University, University of Pennsylvania and University of California, Los Angeles. Early campaigns mirrored advocacy strategies used by groups interacting with National Institutes of Health, Food and Drug Administration, Centers for Medicare & Medicaid Services, Congress of the United States, New York State Legislature, United States Department of Health and Human Services, World Health Organization, European Medicines Agency, Wellcome Trust, European Commission, National Science Foundation, American Academy of Neurology, American Association for the Advancement of Science and other institutional stakeholders.
The organization's mission emphasizes patient-centered advocacy, policy engagement, and clinical trial access, working alongside entities such as U.S. Congress, White House, United States Senate Committee on Health, Education, Labor, and Pensions, United States House Committee on Energy and Commerce, Office of the Commissioner of Food and Drugs, National Organization for Rare Disorders, EURORDIS-Rare Diseases Europe, Global Genes, World Health Organization and international patient networks. It lobbies for legislation, funding priorities at National Institutes of Health, reimbursement policies involving Centers for Medicare & Medicaid Services, and regulatory pathways coordinated with the Food and Drug Administration and European Medicines Agency.
Programmatic work includes family support, care standards dissemination, transition resources, and educational outreach delivered in partnership with clinical centers like Johns Hopkins Hospital, Mayo Clinic, Boston Children's Hospital, Children's Hospital of Philadelphia, Great Ormond Street Hospital, UCSF Benioff Children's Hospital, Boston University School of Medicine, Columbia University Irving Medical Center and university neuromuscular programs at Harvard Medical School, Stanford University School of Medicine, University of Pennsylvania Perelman School of Medicine and University of California, San Francisco. Services include patient registries, genetic counseling linkages, and coordination with rare disease consortia such as those supported by National Institutes of Health networks and philanthropic partners like Wellcome Trust and Gates Foundation.
The organization funds and facilitates research collaborations with academic investigators at Harvard University, Stanford University, University of Oxford, University College London, Massachusetts Institute of Technology, University of California, San Diego, Yale University, Duke University, University of Toronto, McMaster University, Karolinska Institutet, KU Leuven, University of Cambridge and biotech and pharmaceutical partners including Pfizer, Roche, AstraZeneca, Sanofi, Novartis, GlaxoSmithKline, Eli Lilly and Company, Vertex Pharmaceuticals, Biogen, Sarepta Therapeutics, Solid Biosciences, Acceleron Pharmaceuticals, Wave Life Sciences, Amgen and venture capital investors in the life sciences ecosystem. Activities span natural history studies, outcome measure development, gene therapy trials, exon skipping approaches, CRISPR gene editing research at institutions like Broad Institute and regulatory interactions with the Food and Drug Administration and European Medicines Agency for trial design and approval pathways.
Fundraising efforts have included national walks, gala events, community fundraisers, corporate partnerships, and collaborations with philanthropic organizations such as Gates Foundation, Ford Foundation, Rockefeller Foundation, Robert Wood Johnson Foundation and major donors affiliated with universities and hospitals. Signature events connect to wider cultural and sporting institutions, drawing support from entities including New York Yankees, Los Angeles Dodgers, NFL, NBA, Major League Baseball, National Football League, Stanford University Athletics, Harvard Athletics, Yale Bulldogs, Boston Red Sox, Metropolitan Museum of Art, Lincoln Center, Carnegie Hall, Sundance Film Festival, Tribeca Film Festival, Cannes Film Festival, Comic-Con International and corporate sponsors.
The organization is led by an executive team and board of directors comprising family advocates, clinicians, legal professionals, and fundraising experts with ties to institutions such as Columbia University, Harvard University, Stanford University, Johns Hopkins University, Yale University School of Medicine, Mayo Clinic, Cleveland Clinic, Boston Children's Hospital, Children's Hospital of Philadelphia and nonprofit governance advisors who have worked with United Way, American Red Cross, Habitat for Humanity, Bill & Melinda Gates Foundation, W.K. Kellogg Foundation and MacArthur Foundation.
The group's advocacy has influenced research funding priorities at National Institutes of Health, trial access policies at the Food and Drug Administration, and collaborations with pharmaceutical companies leading to advanced candidate therapies. Recognition has come from medical societies including the American Academy of Neurology, patient networks like National Organization for Rare Disorders, awards from civic institutions such as New York City Council and partnerships with academic and philanthropic organizations including Harvard University, Johns Hopkins Hospital, Mayo Clinic, Wellcome Trust, Gates Foundation and Robert Wood Johnson Foundation.
Category:Non-profit organizations based in the United States