Duchenne Alliance Holds Groundbreaking Meeting on Duchenne Muscular Dystrophy, Ignites Revolution in Biomedical Funding
March 11, 2012 - Hotel Boulderado, Boulder, ColoradoThe Duchenne Alliance, a group of nearly 40 international independent foundations focused on Duchenne muscular dystrophy, has taken a groundbreaking step toward winning what it calls "the ultimate race against time."
The leaders of Duchenne non-profit organizations agreed at a meeting last week on the mass implementation of the DUCHENNE DASHBOARD, an innovative research grant management system that will facilitate an unprecedented level of collaboration among the many foundations, patient advocacy groups, and scientists dedicated to finding treatments or cures for Duchenne. Duchenne is the most common and lethal genetic disease diagnosed in childhood. There are currently no life extending treatment options for the over 300,000 males worldwide living with the condition, which usually limits life expectancy to the mid 20s.
The Duchenne Dashboard provides scientists and foundations with a single entry point for proposals to advance Duchenne research. Scientists can use the dashboard to securely submit their projects and member foundations and their scientific advisors can consider and give feedback on them instantly. The tool also enables foundations to quickly collaborate and contribute funds to promising projects.
"The Duchenne Dashboard is going to radically improve and expedite the scientific process in the field of muscular dystrophy. The non-profit foundations came together and set the new standard for effective biomedical funding," said Duchenne Dashboard creator Carlo Rago of OpenOnward. "The platform is now available to all neuromuscular foundations aiming to conquer disease," he said.
The Duchenne Alliance was formed last year to promote better collaboration among the many non-profit organizations around the world committed to conquering Duchenne and improving the lives of those living with this progressive and debilitating muscle wasting condition. "The primary goal of the Alliance is to quickly identify, understand, fund, and implement the most promising research," said Christine McSherry of the Duchenne non-profit Jett Foundation. "The fastest way to success is shared knowledge and collaborative funding," she said.
About the Duchenne Alliance: We are an alliance of independent Duchenne organizations dedicated to advancing our missions to improve quality of life, care, and treatment of those affected by Duchenne. Our goals will be achieved by building trust, sharing knowledge, leveraging resources, and streamlining business practices. Each organization will be held to achieve their own mission as we collectively serve the entire Duchenne community. Please visit www.duchennealliance.org.
About OpenOnward: We employ openscience solutions that empower individuals, groups and societies. Our core products were developed to expedite the entire scientific process by facilitating deeper, more meaningful connections between scientists and society. Our team consists of leaders in biomedical research and open science. We are dedicated to the greater good. Find us on the web at www.openonward.org.
International Duchenne Alliance Annual Conference
March 9th- 10th, 2012 - Hotel Boulderado, Boulder, Colorado"Advancing our missions to improve the quality of life, care and treatment of those affected by Duchenne". - Pledge made by Duchenne Alliance members, March 2011
Greetings International Duchenne Foundations,
Please join us for the first anniversary meeting of the Duchenne Alliance as we harness our collective intelligence and accelerate.
March 9, 2012 - Organizational Updates
11:00am - 9:00pm, "Leveraging Our Unity"
Co-Identify, Co-Understand, and Co-Fund Opportunities. Connect and Speed Science.
Gaining knowledge of projects supported through Alliance member foundations allows additional support opportunities, funding, or other resources needed to push the project forward, faster. Be a part of it - take this opportunity to share with the members what urgently needs funding and what we can do to help.
|11:00 -11:45|| Welcome
|12:00 -1:30|| Session 1 Projects in the Community
Charley's Fund - Halofuginone Update
Parent Project Muscular Dystrophy - TBA
|1:30 - 2:30||Lunch
Duchenne Dashboard Refresh
|2:30 - 4:00|| Session 2 Projects (Continued)
RACE MD - University of Washington
|4:00 - 4:15||Break|
|4:15 - 6:00|| Session 3|
Breakout Groups - Follow up on Presentations
|7:00 - 9:00|| Evening Group Dinner
Duchenne Alliance Governing Discussion
March 10, 2012 - Nonprofit Management and Infrastructure
7:00am - 6:00pm, "Inefficiencies and Inequalities"
Efficient and Effective. Highly Powered Tools that Drive Funds.
As Duchenne Foundation leaders, we need the most highly powered tools available to connect with the public and generate funds. Presentations from highly cited guest speakers will include database resources, tax and financial statements, organizational development, management, and leadership. Our goal is to drive more funds into your organization. We will share tools to address this urgent challenge.
|7:00 - 8:00|| Breakfast
Database Comparisons with Katie Cutler, Jett Foundation
|8:00 - 9:45|| Morning 1 Nonprofit Management Accounting and Financial Statements l
Smith & Smith, CPA
How financial statements and annual reports can help make your nonprofit competitive
|10:00 -12:00|| Morning 2 Nonprofit Management Accounting and Financial Statements ll
Smith & Smith, CPA
What are the essential marketing pieces to a financial statement
|12:00 - 1:00|| Lunch
Fundraising Round Table - Discussion and exchange
|1:00 - 2:45|| Afternoon 1 Nonprofit Organizational Management Series l
Susan Spero, Spero & Company Consultants, LLC
Strategic Planning: what it is and isn't, why it often fails, why it's not optional
|2:45 - 3:00||Break|
|3:00 - 5:00|| Afternoon 2 Nonprofit Organizational Management Series ll
Susan Spero, Spero & Company Consultants, LLC
What to keep in your toolbox - tools for the nonprofit leader
|5:00 - 6:00|| Closing |
Wrap Up and Next Steps
Conference fee: $325 per person, payable to Jett Foundation, 42 Elm Street, Suite 3, Kingston, MA 02364. Includes breakfast and lunch on both days. Includes Alliance member dinner eve of March 9, 2012 at the Hotel Boulderado*Does not include accommodations or transportation.
Hotel Boulderado, be sure to mention your Duchenne Alliance affiliation.
Your Alliance teammates await your participation.
Ryan's Quest provides a travel grant to Johns Hopkins School of Medicine to help recruit patients for the REVERSE-DBMD clinical trial
November 13th, 2011 - Hamilton, NJThe Ryan's Quest Foundation has awarded Kathryn Wagner, MD, PhD, of the Kennedy Krieger Institute, JohnsHopkins School of Medicine, Baltimore, Maryland a $76,500 grant that will ease the financial burden of families who are participating in the REVERSE-DBMD (Revatio for heart disease in Duchenne and Becker Muscular Dystrophies) trial.
Funding for the grant was fully pledged within a week of submission to the Duchenne Dashboard. The funds will be distributed in three rounds as the trial progresses through recruitment milestones. RaceMD, lead by Chris James, provided administrative assistance to expedite the grant process, allowing Ryan's Quest and Johns Hopkins to rapidly move forward with this opportunity. "The Duchenne Alliance is bringing unexpected efficiencies to the scientific process," said Carlo Rago, PhD, a scientific advisor for Ryan's Quest.
"We were very excited to utilize the Dashboard as a way to expedite our funding to Dr. Wagner, as time is not a commodity in Duchenne," said Dave Schultz, Founder of Ryan's Quest.
Recent advances in treatment and care are extending the life expectancy of those affected by Duchenne muscular dystrophy, and preservation of cardiac health is increasingly important as the boys age. PDE5 inhibitors, like Sildenafil, are known to have cardioprotective effects in some models of cardiac damage. Researchers have recently shown that Sildenafil can prevent and reverse the pathological cardiac dysfunction in a mouse model of DMD. Sildenafil is FDA-approved for the treatment of erectile dysfunction and pulmonary hypertension. Two recent reports have described Sildenafil as well tolerated when treating non-Duchenne children and neonates diagnosed with pulmonary hypertension. "These characteristics provide a strong rationale for a clinical trial of Sildenafil in Duchenne," said Carlo Rago, PhD.
Re-purposing of FDA-approved drugs is a hot area in Duchenne research. "We are cautiously optimistic that trials such as Dr. Wagner's will provide a promising treatment in Duchenne muscular dystrophy in the near future. By providing a travel grant, we hope to facilitate recruitment of participants and fill the trial to its capacity, thereby providing reliable and valid outcomes," said Dave Schultz.
Full Press Release: Ryan's Quest Research Press Release.pdf
REVERSE-DBMD clinical trial
Kathryn Wagner, MD, PhD
Duchenne Alliance invites Global Participation at Action Duchenne
November 5th, 2011 - London, EnglandThe Duchenne Alliance took another major step forward presenting at the 9th Annual Duchenne Conference sponsored by Action Duchenne. The international conference for Duchenne muscular dystrophy featured organizations from around the world, representing more than a dozen countries.
"The Duchenne Dashboard was well received at the Action Duchenne Conference in London by leading Duchenne international scientists and clinicians. The Alliance has created a great tool that will enable funders and researchers to work together to accelerate to the clinic new medicines for Duchenne," said Nick Catlin, CEO of Action Duchenne.
Christine McSherry, president of the Jett Foundation, delivered a passionate speech using anessay from Marissa Penrod, president of Team Joseph, as a guide to describe the advantages of collaboration through the Alliance and the Dashboard software. Christine described the Dashboard as a conduit to expedite the workflow of scientists and foundations by streamlining the submission, review and funding of proposals dedicated to advancing research, treatment or care for Duchenne.
Carlo Rago, PhD, CEO of OpenOnward,followed with an open invitation to participate on the Dashboard and he is working closely with many scientists to prepare and load proposals for review and funding by Alliance members. The primary goal of the Dashboard is to host a complete list of current research opportunities and facilitate an active review process that leads to better funding decisions in the Duchenne community. "The facility to crowd-source scientific advisors provides all participating Alliance members access to this limited resource," said Carlo Rago.
The next critical meeting for the Duchenne Alliance will take place in early March in Boulder, CO and will be announced shortly.
audio available: Alliance and Dashboard at Action Duchenne Full Press Release:
Duchenne Alliance: An Urgent Call to Action
July 10th 2011 - Baltimore, MD
This meeting served as an open invitation and broad overview to encourage all Foundations to join the Alliance. Members of the public and scientific enterprise were introduced to the Duchenne Dashboard as a new resource that will enhance interactions within the Duchenne community.
PPMD Duchenne Connect Meeting, Marriott Waterfront
Welcome: Dave Schultz, Ryan's Quest
Call to Action: Christine McSherry, Jett Foundation
Purpose and Mission: Tonya Dreher, Hope for Gus
Collaboration: Marissa Penrod, Team Joseph
Efficiency: Chris James, Race MD
Duchenne Dashboard: Carlo Rago, OpenOnward
Questions: Open to Audience
Duchenne Alliance: Optimizing our Opportunity
July 7th 2011 - Baltimore, MD
A presentation of the Duchenne Dashboard by OpenOnward and members of the Duchenne Alliance lead to an open discussion that identified several action items.
Oceanaire Seafood Room
The "Epic" Meeting at the Wentworth by the Sea