Administrative Core

Virginia Commonwealth University, Richmond VA

Investigators

Abstract

Modified Project Summary/Abstract Section Our Center seeks to achieve three major goals: 1) Addressing, using a team science approach, the major gaps in therapeutic development with a preparing the pediatric population as well as identifying genetic modifiers of response to therapies; 2) Promoting therapeutic development external to the Center; 3) Fostering the development of junior scientists with a focus on therapeutic development in Myotonic Dystrophy (DM). The completion of these goals requires engagement of multiple stakeholders, including the multiple Center investigators and sites, academic and industry partners, patient advocacy groups and (most importantly) patients with DM. The Administrative Core will seek to learn from lessons interacting in a virtual environment that were developed during the recent public health emergency. This Core will bring these stakeholders together virtually and seek to create project milestones that allow for completion of the overall objectives. This Core will seek to accomplish these goals through the three complementary approaches. Firstly, the investigators will create the collaborative structure to facilitate the completion of individual projects. This structure will utilize remote video conference platforms, such as Zoom, and in real time messaging tools, such as Teams, to create a virtual Center environment that will allow a clear framework for communication. This will be coupled with in person annual meetings. Secondly, the investigators will link these virtual communication platforms with project management software to allow for the Executive Committee, External Advisory Committee, and Patient Engagement Committee to track progress and adjust project milestones accordingly. Finally, the investigators will create a robust and interactive website that would serve as an entry into the Center, including access to resources and materials. This would be the cornerstone of a larger effort to integrate the Center into the larger DM scientific research community, the DM patient community, and the Wellstone Center community. As part of our effort to bring new scientists into Muscular Dystrophy, the executive committee members will pair with new investigators to provide mentorship and further engage these scientists in myotonic dystrophy. To accomplish these goals, the investigators will leverage the prior experience of the research teams in successfully performing multicenter projects. The Center is fortunate to have assemble investigators with a long history of collaboration and an understanding of the team science approach. The Center will leverage all of these resources to achieve our shared goal of accelerating disease modifying therapies for the myotonic dystrophies and including those populations (e.g., children) who are not currently able to access these therapeutic trials. Modified Project Summary/Abstract Section Our Center seeks to achieve three major goals: 1) Addressing, using a team science approach, the major gaps in therapeutic development with a preparing the pediatric population as well as identifying genetic modifiers of response to therapies; 2) Promoting therapeutic development external to the Center; 3) Fostering the development of junior scientists with a focus on therapeutic development in Myotonic Dystrophy (DM). The completion of these goals requires engagement of multiple stakeholders, including the multiple Center investigators and sites, academic and industry partners, patient advocacy groups and (most importantly) patients with DM. The Administrative Core will seek to learn from lessons interacting in a virtual environment that were developed during the recent public health emergency. This Core will bring these stakeholders together virtually and seek to create project milestones that allow for completion of the overall objectives. This Core will seek to accomplish these goals through the three complementary approaches. Firstly, the investigators will create the collaborative structure to facilitate the completion of individual projects. This structure will utilize remote video conference platforms, such as Zoom, and in real time messaging tools, such as Teams, to create a virtual Center environment that will allow a clear framework for communication. This will be coupled with in person annual meetings. Secondly, the investigators will link these virtual communication platforms with project management software to allow for the Executive Committee, External Advisory Committee, and Patient Engagement Committee to track progress and adjust project milestones accordingly. Finally, the investigators will create a robust and interactive website that would serve as an entry into the Center, including access to resources and materials. This would be the cornerstone of a larger effort to integrate the Center into the larger DM scientific research community, the DM patient community, and the Wellstone Center community. As part of our effort to bring new scientists into Muscular Dystrophy, the executive committee members will pair with new investigators to provide mentorship and further engage these scientists in myotonic dystrophy. To accomplish these goals, the investigators will leverage the prior experience of the research teams in successfully performing multicenter projects. The Center is fortunate to have assemble investigators with a long history of collaboration and an understanding of the team science approach. The Center will leverage all of these resources to achieve our shared goal of accelerating disease modifying therapies for the myotonic dystrophies and including those populations (e.g., children) who are not currently able to access these therapeutic trials.