STRENGTH HAPPENS TOGETHER: PPMD’S CORPORATE & ORGANIZATIONAL PARTNERS
For over two decades, PPMD has funded projects we feel are the most impactful and will treat as many people in our community as quickly and effectively as possible. We pride ourselves on being intricately involved at all levels of the therapy development pipeline and recognize that the progress made is inextricably linked to our crucial advocacy work. We are dedicated to moving all potential therapies forward. We invest funds raised as quickly and efficiently as possible to maintain our comprehensive approach.
We know that no one organization or company will end Duchenne. That’s why we are grateful to all of our collaborators who, by joining forces with PPMD and the Duchenne community, make us all stronger. PPMD will accept corporate support so long as it is in accordance with our Corporate Relations Policy (download).
Duchenne Drug Development Roundtable
PPMD’s Drug Development Roundtable (DDDR) is comprised of the majority of industry partners engaged in developing therapeutics for Duchenne, that come together in the pre-competitive space to address shared challenges and opportunities in Duchenne therapy development. The goal is to accelerate the development of meaningful treatments for Duchenne muscular dystrophy through open discussion and collaborative engagements. Membership is open to all sponsors within the Duchenne space; an annual fee structure to support DDDR meetings and collaborative DDDR activities is required of participants.
2019 Drug Development Roundtable Members
2019 Drug Development Roundtable Partners
Strength in Inspiration: Corporate Sponsors
Our corporate sponsors work closely with us to achieve our mission. These Sponsors invest in our key pillars: Research, Advocacy, Care, and Education through relevant projects, efforts, initiatives, programs, and more. We are proud to recognize the mission support of our sponsors and thank them for joining us as we work to end Duchenne.
Strength in Collaboration: Project Partners
As the catalyst to move research forward, PPMD is dedicated to promoting the patient voice in advocating for developing Duchenne therapies and insisting on patient involvement in studies and trials. We are proud to recognize our project partners who, like us, believe that patients need a voice in the fight to end Duchenne.
Newborn Screening Pilot Program in New York State
Launched by our national effort to build a Newborn Screening (NBS) infrastructure for Duchenne in the U.S. aimed at developing the evidence to support Duchenne NBS, in 2018, we proudly introduced initiation of the Duchenne Newborn Screening Pilot in New York State.
The pilot is being funded and led by a consortium of Duchenne industry partners with a commitment to early diagnosis and intervention in Duchenne.
Decode Duchenne 2019
PPMD Care Meeting Sponsors
End Duchenne Tour Sponsors
Patient Preference Study Partnerships and Publications
- Engaging Patients and Caregivers in Prioritizing Symptoms Impacting Quality of Life for Duchenne and Becker Muscular Dystrophy (download)
- Patient-Centered Benefit–Risk Assessment in Duchenne Muscular Dystrophy – Meaningful pulmonary outcomes (download)
Pfizer & EveryLife Foundation
- How Do Members of the Duchenne and Becker Muscular Dystrophy Community Perceive a Discrete-Choice Experiment Incorporating Uncertain Treatment Benefit? An Application of Research as an Event (download)
- Gene therapy as a potential therapeutic option for Duchenne muscular dystrophy: A qualitative preference study of patients and parents (download)
- Priorities when deciding on participation in early-phase gene therapy trials for Duchenne muscular dystrophy: a best– worst scaling experiment in caregivers and adult patients (download)
Annual Connect Conference
For information on PPMD’s Annual Conference and sponsors, please visit our Conference page.
Strength in Education: Engagement Partners
Knowledge is power in the fight to end Duchenne, and these partners help PPMD bring important information and critical updates to the Duchenne community, as well as the broader rare disease community.
PPMD Corporate Partner Income Breakdown
Support from our partners drives our programs and initiatives forward. In 2018*, support from our corporate partners totaled $3,411,209 – 36% of our total income for that year.
*latest available audited financial statement
Strength in Network: Organizational Partners
PPMD maintains formal and informal partnerships with organizations around the nation and globe who share a similar mission, tactics, and ideology for the purposes of cooperation on research, sharing data, and raising awareness for the entire rare disease community.
- Clinical Trials Transformation Initiative, Patient Executive Committee
- Clinical Trials Transformation Initiative, Patient Leadership Council
- DIA Master Protocol Workshop Planning Committee 2018, 2019
- FasterCures Intellectual Property Intersection Web Site Advisory Group member
- Faster Cures Nonprofit Council
- Friedreich’s Ataxia Research Alliance (FARA) Registry Steering Committee member
- Geisinger Health System (IRB of record for The Duchenne Registry)
- Health Research Alliance (HRA) Board of Directors
- Imaging DMD Steering Committee
- Institute of Medicine (IOM) Committee on Pediatric Studies
- National Institute of Neurological Disorders and Stroke (NINDS), National Advisory Council
- National Society of Genetic Counselors Neurogenetics Special Interest Group member
- New York University School of Medicine, Compassionate-Use Working Group
- PCORnet® (The Duchenne Registry was a Network Partner in Phase 1 & 2 of PCORnet®, the National Patient-Centered Clinical Research Network)
- RAND Corporation
- RTI International
- TREAT-NMD’s Advisory Committee for Therapeutics, ad hoc member
- TREAT-NMD’s Global Data Oversight Committee member
- UCLA Center for Duchenne Muscular Dystrophy
- University of Arizona – School of Mind, Brain and Behavior, Advisory Board
- VeloCTTI Master Protocol Project Team Lead
- 2018 Extended Access Summit, Steering Committee
- Alliance for a Stronger FDA
- Biotechnology Innovation Organization (BIO) Patient Advocacy Group
- Center for Patient-Driven Value Assessment Steering Committee
- Department of Defense (DOD) Chair of Congressionally Directed Medical Research Program in Duchenne
- European Community Advisory Board, Chair
- EveryLife Rare Hub partner
- Friends of NCBDDD (CDC)
- Friends of NICHD (Eunice Kennedy Shriver National Institute for Child Health and Human Development)
- Global Genes Project
- Muscular Dystrophy Coordinating committee (MDCC)
- National Health Council Board of Directors
- National Health Council Medical Innovation Action Team
- National Health Council Patient Centered Outcomes Research Institute (PCORI) Value Workgroup
- National Institutes for Arthritis Musculoskeletal Disease and Skin (NIAMS) Patient Advocacy Coalition
- Rare Disease Legislative Advocates (RDLA)
- World Duchenne Organization (formerly UPPMD)
- American Child Neurology Nurses Nominating Committee, chairman
- CDC Care Considerations Implementation and Evaluation Committee
- CDC Care Considerations Steering Committee
- CDC Working Group on Quality of Life Models and Measures for People with Muscular Dystrophy and Other Significant Mobility Limitations
- Office of Rare Disease Research Global Rare Diseases (Patient) Registry and Data Repository (GRDR), member of selection committee in 2012
- PPMD staff and consulting professionals now include four experienced healthcare providers: 2 board-certified genetic counselors, a registered nurse, and a board-certified pediatric nurse practitioner
- American Academy of Pediatrics, Motor Delay Plain Language Project, Expert Collaborator
- American Academy of Pediatrics, Steering Committee
- PPMD genetic counselors regularly assist with graduate student thesis projects (MS and PhD) involving Duchenne, including the study design, implementation, recruitment, and advisory committee roles
- PPMD staff regularly provides trainings for healthcare providers, educators, researchers, families, and other stakeholders.
- Rare Disease Task Force, PCORnet®, Co-Chair