CureDuchenne
Our vision is our name: To Cure Duchenne
Programs and results
What we aim to solve
Our programs
What are the organization's current programs, how do they measure success, and who do the programs serve?
CureDuchenne Research
CureDuchenne is the leading non-profit funding organization for Duchenne research. Our focus on high-impact research has accelerated progress toward new therapeutic approaches as well as capitalized on existing technology, like CRISPR-CAS9 gene-editing that has changed the research landscape. Today CureDuchenne has a global reach and funds multiple Duchenne and platform technology projects each year. Like our early support of research in dystrophin-restoration, we are again taking a major step toward a future cure. We are building the world’s first Duchenne and Becker muscular dystrophy biobank, to give researchers a 360-degree view of life with DMD. CureDuchenne Link uses custom technology and builds on our nation-wide community network. It is hosted in one all-encompassing platform using industry-standard terminology compatible with other data sets. These securely coded bio-samples and survey data are then made accessible to qualified researchers.
CureDuchenne Cares
CureDuchenne understands the needs of the patient community. Our healthcare experts, in-house and collaborators, are current on all treatment options, from physical therapy to therapeutics and clinical trials. These experts counsel and educate Duchenne patients and families on best care options and bring the Duchenne medical community together to improve standard of care. Our interactive education and outreach programs provide information, resources, and best practices for managing the challenges of Duchenne to help improve overall quality of life for families across the country. From advocacy for neonatal screening to provision of a directory of neurologists with Duchenne expertise, to a Physical Therapist certification program, our aim is to ensure expert care for families. Our work has expanded globally with Duchenne care providers and families across North and South America, Africa, Europe, Asia, and Australia, requesting our expertise and services.
Where we work
External reviews
Our results
How does this organization measure their results? It's a hard question but an important one.
Number of public events held to further mission
This metric is no longer tracked.Totals By Year
Type of Metric
Output - describing our activities and reach
Direction of Success
Increasing
Number of health education trainings conducted
This metric is no longer tracked.Totals By Year
Type of Metric
Output - describing our activities and reach
Direction of Success
Increasing
Number of rallies/events/conferences/lectures held to further mission
This metric is no longer tracked.Totals By Year
Type of Metric
Output - describing our activities and reach
Direction of Success
Increasing
Number of community events or trainings held and attendance
This metric is no longer tracked.Totals By Year
Type of Metric
Output - describing our activities and reach
Direction of Success
Increasing
Context Notes
1300 attendees
Number of individuals attending community events or trainings
This metric is no longer tracked.Totals By Year
Type of Metric
Output - describing our activities and reach
Direction of Success
Increasing
Number of attendees present at rallies/events
This metric is no longer tracked.Totals By Year
Type of Metric
Output - describing our activities and reach
Direction of Success
Increasing
Goals & Strategy
Learn about the organization's key goals, strategies, capabilities, and progress.
Charting impact
Four powerful questions that require reflection about what really matters - results.
What is the organization aiming to accomplish?
We are committed to improving the lives of everyone affected by Duchenne through accelerating research to find the cure, improving care and empowering the Duchenne community.
What are the organization's key strategies for making this happen?
From the start CureDuchenne went directly to the scientific experts in the field of Duchenne to help guide our funding of potential therapies to treat and ultimately cure Duchenne. CureDuchenne’s Scientific Advisory Board consists of the leading scientists in the world helping us determine the most viable research projects, accelerate the clinical trial process and bring potential life-saving drugs to help this generation of Duchenne boys. Our scientific board in partnership with our in-house Chief Scientific Officer work to identify new cutting edge research with the ability to quickly progress to human clinical trials.
What are the organization's capabilities for doing this?
We recently created an initiative called CureDuchenne Ventures. CureDuchenne Ventures funding strategy includes identifying drug targets and programs that exhibit the potential to advance rapidly to proof of concept studies. The goal of CureDuchenne Ventures is to provide substantial funding to several research projects over the next five years that will serve as a catalyst to drive significant investment from biotech and pharmaceutical companies and venture philanthropists. CureDuchenne has adopted a broad research strategy to identify and fund novel therapies that target specific components of the disease. We expect that as these projects progress to clinical settings, they will dramatically improve the prognosis for children diagnosed with Duchenne. Each project will allow CureDuchenne to receive a return based on success which will be reinvested in future projects creating a self-sustaining research budget.
What have they accomplished so far and what's next?
CureDuchenne has a history of success and has funded seven research projects that have advanced into human clinical trials. Two of our funded projects are now in late-stage Phase II and Phase III clinical trials around the world and could become the first drugs to be approved for the treatment of Duchenne.
We are waiting for the first FDA approval of a Duchenne muscular dystrophy drug, and although close there are no approved drugs today.
How we listen
Seeking feedback from people served makes programs more responsive and effective. Here’s how this organization is listening.
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How is your organization using feedback from the people you serve?
To identify and remedy poor client service experiences, To identify bright spots and enhance positive service experiences, To make fundamental changes to our programs and/or operations, To inform the development of new programs/projects, To strengthen relationships with the people we serve, To understand people's needs and how we can help them achieve their goals
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Which of the following feedback practices does your organization routinely carry out?
We collect feedback from the people we serve at least annually, We take steps to get feedback from marginalized or under-represented people, We aim to collect feedback from as many people we serve as possible, We take steps to ensure people feel comfortable being honest with us, We look for patterns in feedback based on demographics (e.g., race, age, gender, etc.), We look for patterns in feedback based on people’s interactions with us (e.g., site, frequency of service, etc.), We engage the people who provide feedback in looking for ways we can improve in response, We act on the feedback we receive, We share the feedback we received with the people we serve, We tell the people who gave us feedback how we acted on their feedback, We ask the people who gave us feedback how well they think we responded
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What challenges does the organization face when collecting feedback?
We don't have any major challenges to collecting feedback
Financials
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Operations
The people, governance practices, and partners that make the organization tick.
Connect with nonprofit leaders
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- Analyze a variety of pre-calculated financial metrics
- Access beautifully interactive analysis and comparison tools
- Compare nonprofit financials to similar organizations
Want to see how you can enhance your nonprofit research and unlock more insights? Learn More about GuideStar Pro.
Connect with nonprofit leaders
SubscribeBuild relationships with key people who manage and lead nonprofit organizations with GuideStar Pro. Try a low commitment monthly plan today.
- Analyze a variety of pre-calculated financial metrics
- Access beautifully interactive analysis and comparison tools
- Compare nonprofit financials to similar organizations
Want to see how you can enhance your nonprofit research and unlock more insights? Learn More about GuideStar Pro.
CureDuchenne
Board of directorsas of 11/20/2023
Jeffrey Goffman
Debra Miller
CureDuchenne
Paul Miller
Go Fit Foods
Ryan Williams
Athletes First
Elaine Wu
WHGC, P.L.C.
TJ Farnsworth
SightLine Health, LLC
Sarah McElroy
South Coast Repertory Theater
Jeffrey Goffman
Integrated Oncology Network, LLC
Martin Lehr
Context Therapeutics
David Maclnnis
Advent Children’s Hospital Orlando
Peter Strelow
PIMCO
Tim Sullivan
ActivePDF, Inc.
Dana White
Hyundai Motor North America
Board leadership practices
GuideStar worked with BoardSource, the national leader in nonprofit board leadership and governance, to create this section.
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Board orientation and education
Does the board conduct a formal orientation for new board members and require all board members to sign a written agreement regarding their roles, responsibilities, and expectations? Yes -
CEO oversight
Has the board conducted a formal, written assessment of the chief executive within the past year ? Yes -
Ethics and transparency
Have the board and senior staff reviewed the conflict-of-interest policy and completed and signed disclosure statements in the past year? Yes -
Board composition
Does the board ensure an inclusive board member recruitment process that results in diversity of thought and leadership? Yes -
Board performance
Has the board conducted a formal, written self-assessment of its performance within the past three years? Yes
Organizational demographics
Who works and leads organizations that serve our diverse communities? Candid partnered with CHANGE Philanthropy on this demographic section.
Leadership
The organization's leader identifies as:
Race & ethnicity
Gender identity
Transgender Identity
Sexual orientation
No data
Disability
No data
Equity strategies
Last updated: 10/31/2023GuideStar partnered with Equity in the Center - an organization that works to shift mindsets, practices, and systems to increase racial equity - to create this section. Learn more
- We review compensation data across the organization (and by staff levels) to identify disparities by race.
- We ask team members to identify racial disparities in their programs and / or portfolios.
- We analyze disaggregated data and root causes of race disparities that impact the organization's programs, portfolios, and the populations served.
- We disaggregate data to adjust programming goals to keep pace with changing needs of the communities we support.
- We employ non-traditional ways of gathering feedback on programs and trainings, which may include interviews, roundtables, and external reviews with/by community stakeholders.
- We disaggregate data by demographics, including race, in every policy and program measured.
- We have long-term strategic plans and measurable goals for creating a culture such that one’s race identity has no influence on how they fare within the organization.
- We have a promotion process that anticipates and mitigates implicit and explicit biases about people of color serving in leadership positions.
- We have community representation at the board level, either on the board itself or through a community advisory board.
- We help senior leadership understand how to be inclusive leaders with learning approaches that emphasize reflection, iteration, and adaptability.