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The chart above shows, graphically, where the Duchenne field is a each stage of the drug development process and what knowledge and infrastructure is already in place. Several mouse models exist for the disease, the most commonly used of which is the mdx mouse, which has a mutation that causes the dystrophin protein to stop being made prematurely.
The societal burden of Duchenne is not well understood in the US and Europe, although relatively current data exists in Australia.
Drug development programs do exist both in industry in large and small companies and in academic laboratories. A regulatory path for new therapies for Duchenne is in progress through the pioneering efforts of PTC Therapeutics, the first company to attempt to get a drug approved for Duchenne, and followed now by several other companies. Multiple experienced clinical research networks exist in the US and Europe but the individual networks lack cohesion and there is some duplication of effort. The European government-funded TREAT-NMD consortium has sponsored a global patient registry that receives curated core data elements from individual Duchenne patient registries all over the world. Remaining infrastructure needs include standardized genotyping, validated biomarkers, and earlier diagnosis and identification of children in rural or inner city areas.
To learn more about how PPMD will address gaps in resources, see our Programs and Priorities. Columbus, OH — Earlier this summer, I had a great conversation with Matt Cash, an ambitious innovator who has designed everything from better sales tools to more addictive sports fantasy games. Working with colleagues around the world, we uncovered 11 healthcare trends that are changing the world of pharma sales – from doctors becoming less central to healthcare to a new global center of opportunity in Asia-Pac to evolving multi-media expectations from physicians.
Soon this report will require registration to view, but we’re excited to share it first with HxP readers. Want HXP Updates?To begin receiving HXP updates, please fill out the required information below. Welcome to Columbia University Human Resources. We are a department committed to operational excellence, ethical leadership and expanding the value and effectiveness of the services we offer to support the University's mission and the community in which we live and work.


Aspirations to Achievement: Men of Color and Community Colleges, a report from the Center for Community College Student Engagement, builds on the growing body of research about the experiences of men of color in higher education.
Donate to Project MALES!Your donation to Project MALES will help support the mission and work of this exciting new initiative. The symptoms in the dogs more closely mimic the human version of the disease and therapies with challenging delivery issues (like gene therapy) are often tested in these dogs. This information is useful in calculating what insurers would be likely to pay for therapies and in advocacy efforts. In addition to companies focused on Duchenne specifically, many of the large pharma have programs focused on age-related (sarcopenia) and illness-related (cachexia) muscle wasting that may have applicability to Duchenne. As soon as one drug has been successfully approved, that process should provide a road map for future submissions to regulatory agencies.
A workshop held in conjunction with PPMD's Annual Connect Conference identified the need for an outreach mechanism to clinics with less experience in conducting trials. In the US there are two Duchenne registries (including PPMD's DuchenneConnect), each with close to 2,000 individual entries, and a third is planned.
Although useful as a minimal standard of care, room to improve on this first effort remains. In addition, few modifier genes for Duchenne have been identified to date, but modifiers could add additional drug targets to the pipeline. He started the conversation with a big challenge: We have to find a better way to arm the salesforce.
If we really got close to the trends changing the salesforce, what new experiences and new best practices could we uncover?
Looking at that landscape led us to new best practices for pharma sales as well as five emerging opportunities – from on-the-go learning to a packaged sale that brings together diagnostics with treatment and proven support.
We hope you’ll challenge our thinking and help us continue this conversation about just what kind of experience we should be creating for the frontlines.


I've worked with Fortune 1000 companies to craft their digital, mobile, social and CRM strategies for 15 years. The report includes an analysis of student engagement survey results for over 145,000 male community college students and provides information for community colleges on how to get better outcomes for these students. Victor Saenz, executive director of Project MALES and associate professor at the University of Texas at Austin, served on the advisory committee for this report. Your contribution will assist in encouraging young Latino male students at UT-Austin--as well as middle school and high school youth throughout Central Texas--to raise their expectations, to excel academically, and to become more responsible and engaged citizens in our communities.
More work needs to be done to develop new qualified endpoints and particularly those that can be used in non-ambulatory subjects. Although all will feed into to the TREAT-NMD Global patient registry, the existence of multiple registries duplicates resources and causes confusion among families.
Well-adopted standards of care not only help improve care, but also serve to reduce variability in clinical testing.
At GSW, I founded an innovation practice fueled by the zeitgeist; our team uncovers consumer trends and puts them to work to break through the barriers of old thinking, ask “what could be” for clients, and create entirely new possibilities for healthcare experience.
The Center conducted six focus groups with faculty and staff, and more than 30 student focus groups with Latino, Black and White men at community colleges and the Phi Theta Kappa Honor Society national convention.
Project MALES is an initiative of DDCE and UT-Austin, and your donation is tax deductible to the fullest extent allowable by law. A mouse that lacks both dystrophin and a similar protein called utrophin is more severely affected than the mdx mouse and used to test some interventions.



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