# Administrative Core

> **NIH NIH P50** · UNIVERSITY OF ROCHESTER · 2021 · $93,878

## Abstract

The myotonic dystrophies (DM) are the most common group of adult muscular dystrophies. The core
characteristics of myotonic dystrophy type 1 (DM1) and type 2 (DM2) are dominant inheritance, myotonia,
cataracts, muscle weakness, and multi-system manifestations that are often severe and disabling. Rapid
advancements are occurring in regard to new technology for communicating with patients, new information
about disease progression and biomarkers of severity, along with new approaches to therapy. Several new
initiatives have been proposed to maintain and enhance our Administrative Core to help meet these needs and
leverage support amongst our Projects and Cores, collaborators, and moreover, patients and their families. The
mission of our Wellstone Center is to promote translational research that leads to transformative treatments
for DM1 and DM2. The main goals of the Administrative Core of our Wellstone Center are to coordinate and
oversee funding, regulations, and communication amongst and between all stakeholders currently supporting
and collaborating with us (advocacy groups, foundations, pharmaceutical companies, academia, NIH, and
patients). Specifically, Aim 1 is designed to maintain leadership and manage funds, infrastructure, and
reporting of activities in our proposed Wellstone Center. Leadership is centralized on principal investigators at
the University of Rochester and University of Florida, who have collaborated for 20 years and made significant
accomplishments in the DM field. We will use the necessary tools of communication and schedule meetings to
receive guidance of our external Center Advisory Committee, which consists of leaders in translational science,
clinical care, and patient advocacy. In addition, we will provide oversight to assure all regulatory guidelines,
privacy, and safety precautions are followed to recruit and enroll human subjects in proposed projects. Aim 2 is
designed to maintain the synergy with our Wellstone Cores, Projects, and outside collaborators, in order to
promote the use of the Shared Scientific Resource Core. We will oversee data and resource sharing plans from
resources collected within our Wellstone Projects and the Scientific Core, in order to share information and
collaborate with the broad community of physicians and patients interested in the pathophysiology and
treatment of DM. Aim 3 seeks to develop educational outreach with patients, advocacy groups, care providers,
and researchers. We will build upon existing resources (printed and electronic newsletters, social media,
conferences, and videos) to enhance “two-way” communication between investigators and patients about the
diagnosis, clinical management, and research related to DM. In addition, the Administrative Core will bring
together the robust assets that are available within the Medical Center, such as, our Clinical & Translational
Sciences Institute and our Departmental resources, to facilitate the activities of our Wellstone projects a...

## Key facts

- **NIH application ID:** 10237265
- **Project number:** 5P50NS048843-19
- **Recipient organization:** UNIVERSITY OF ROCHESTER
- **Principal Investigator:** CHARLES A THORNTON
- **Activity code:** P50 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2021
- **Award amount:** $93,878
- **Award type:** 5
- **Project period:** 2003-09-30 → 2023-08-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10237265

## Citation

> US National Institutes of Health, RePORTER application 10237265, Administrative Core (5P50NS048843-19). Retrieved via AI Analytics 2026-05-24 from https://api.ai-analytics.org/grant/nih/10237265. Licensed CC0.

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