The pace of scientific advancements, development of new clinical guidelines, and """"""""instant access"""""""" to web based data and information is rapidly changing and often requires flexible guidance and oversight. Such oversight is crucial as experimental therapies may outpace the resources available to translate these findings into clinical trials, especially in myotonic dystrophy type 1 (DM1). The main goals of the Administrative Core of our Wellstone Center are to coordinate and oversee seamless communication of all activities in the renewal application of our Wellstone Center.
The specific aims of the Administrative Core are to coordinate and integrate the components &activities as well as the utilization of funds by the 2 scientific projects, the Scientific Resources Core and the Training &Educational Core that comprise our Wellstone Center. The Administrative Core will play an especially important role in coordinating the interactions between the newly established Myotonic Dystrophy Clinical Research Network (DM CRN) that consists of 5 sites (Ohio State University, Stanford University, University of Florida, University of Kansas, and University of Rochester). We have proposed collaborative studies within the DM CRN to validate endpoints, biomarkers, and patient reported outcomes as described in Project 1. Our Administrative Core gains vitality and effectiveness from prior collaborations with each of these sites and locally, from the experience of our staff and investigators, who have coordinated activities over the past two cycles of our Wellstone Center (2003-2013). The necessary communication network, knowledge of storage and confidentiality requirements, data tracking software, and computer facilities to operate a large-scale, multi-center, NIH funded, project are already in place. We will enhance our communications with various committees, both internally and externally, to facilitate smooth operations of our Wellstone activities and interactions with the DM CRN. In addition, the Administrative Core will bring together the robust assets that are available within the Medical Center, such as, the recently funded Clinical &Translational Sciences Institute and our Departmental resources, to facilitate the activities of our scientific projects and cores.

Public Health Relevance

The monitoring and communication responsibilities of our Administrative Core are vital to implement the activities proposed in our new Wellstone Center in order to prepare for promising experimental therapies in myotonic dystrophy type 1. Our administrate goals are synergized by collaborations within and outside the Wellstone Network, resources of our Medical Center, and long-standing interactions with patients.

Agency
National Institute of Health (NIH)
Institute
National Institute of Neurological Disorders and Stroke (NINDS)
Type
Specialized Center--Cooperative Agreements (U54)
Project #
5U54NS048843-12
Application #
8733762
Study Section
Special Emphasis Panel (ZNS1-SRB-S)
Project Start
Project End
Budget Start
2014-09-01
Budget End
2015-08-31
Support Year
12
Fiscal Year
2014
Total Cost
$104,749
Indirect Cost
$36,509
Name
University of Rochester
Department
Type
DUNS #
041294109
City
Rochester
State
NY
Country
United States
Zip Code
14627
Johnson, Nicholas E; Ekstrom, Anne-Berit; Campbell, Craig et al. (2016) Parent-reported multi-national study of the impact of congenital and childhood onset myotonic dystrophy. Dev Med Child Neurol 58:698-705
Slean, Meghan M; Panigrahi, Gagan B; Castel, Arturo López et al. (2016) Absence of MutSβ leads to the formation of slipped-DNA for CTG/CAG contractions at primate replication forks. DNA Repair (Amst) 42:107-18
Fitzgerald, Bryan P; Conn, Kelly M; Smith, Joanne et al. (2016) Medication adherence in patients with myotonic dystrophy and facioscapulohumeral muscular dystrophy. J Neurol 263:2528-2537
Heatwole, Chad; Bode, Rita; Johnson, Nicholas E et al. (2016) Myotonic dystrophy health index: Correlations with clinical tests and patient function. Muscle Nerve 53:183-90
Gloss, David; Moxley 3rd, Richard T; Ashwal, Stephen et al. (2016) Practice guideline update summary: Corticosteroid treatment of Duchenne muscular dystrophy: Report of the Guideline Development Subcommittee of the American Academy of Neurology. Neurology 86:465-72
Heatwole, Chad; Johnson, Nicholas; Bode, Rita et al. (2015) Patient-Reported Impact of Symptoms in Myotonic Dystrophy Type 2 (PRISM-2). Neurology 85:2136-46
Yadava, Ramesh S; Foff, Erin P; Yu, Qing et al. (2015) TWEAK/Fn14, a pathway and novel therapeutic target in myotonic dystrophy. Hum Mol Genet 24:2035-48
Pandey, Sanjay K; Wheeler, Thurman M; Justice, Samantha L et al. (2015) Identification and characterization of modified antisense oligonucleotides targeting DMPK in mice and nonhuman primates for the treatment of myotonic dystrophy type 1. J Pharmacol Exp Ther 355:329-40
Wojtkowiak-Szlachcic, Agnieszka; Taylor, Katarzyna; Stepniak-Konieczna, Ewa et al. (2015) Short antisense-locked nucleic acids (all-LNAs) correct alternative splicing abnormalities in myotonic dystrophy. Nucleic Acids Res 43:3318-31
Smith, Amanda E; McMullen, Kara; Jensen, Mark P et al. (2014) Symptom burden in persons with myotonic and facioscapulohumeral muscular dystrophy. Am J Phys Med Rehabil 93:387-95

Showing the most recent 10 out of 74 publications