Our goal for the RDCRC Administrative Unit is to provide an optimal environment to conduct carefully developed and monitored clinical and translational studies that will increase our knowledge about the causes of rare autonomic diseases, and facilitate the development of therapies than can make a significant difference in the lives of patients with these diseases. Ultimately we hope to be on a trajectory to find cures for them. Our consortium benefits greatly from the support of our patients and the Patient Advocate Group (PAG) community to gain insight into the needs and aspirations of that community from the patient's and the caregiver's perspective. The PIs of this RDCRC renewal have a long relationship with the PAGs. The initial PAG for Multiple System Atrophy (MSA) was founded by Dorothy Trainor-Kingsbury, Dr. Sterling Edwards, and Dr. Robertson in 1989. In the subsequent 25 years, there have been annual meetings for patients throughout the United States, and Dr. Robertson has served continuously on the board for this PAG, now known as the MSA Coalition. By attention to the perspective of the PAGs we can tailor our approaches toward both needs of patients and the science that can be addressed to yield the cures that we seek. The Administrative Unit brings RDCRC researchers, PAGs, and the ORDR onto common ground and enables us to work to find a cause and eventually an effective therapy for our patients. We also perceive that in an era of limited funding, we must share the responsibility, to the extent possible, of leveraging the support ORDR and NCATs can provide for the studies we need. In that context we have sought leveraging funds and, in the best current estimate, believe they will reach close to $1.5 million. The administrative structures we have in place should lead to successful biomarker and therapeutic investigations that will improve the health of our patients.

Agency
National Institute of Health (NIH)
Institute
National Institute of Neurological Disorders and Stroke (NINDS)
Type
Specialized Center--Cooperative Agreements (U54)
Project #
6U54NS065736-08
Application #
9146408
Study Section
Special Emphasis Panel (ZNS1-SRB-S)
Project Start
Project End
Budget Start
2016-07-01
Budget End
2017-06-30
Support Year
8
Fiscal Year
2016
Total Cost
$189,700
Indirect Cost
$68,872
Name
Vanderbilt University Medical Center
Department
Type
DUNS #
079917897
City
Nashville
State
TN
Country
United States
Zip Code
37232
Norcliffe-Kaufmann, Lucy; Kaufmann, Horacio; Palma, Jose-Alberto et al. (2018) Orthostatic heart rate changes in patients with autonomic failure caused by neurodegenerative synucleinopathies. Ann Neurol 83:522-531
Raj, Satish R; Robertson, David (2018) Moving from the present to the future of Postural Tachycardia Syndrome - What we need. Auton Neurosci 215:126-128
Coon, Elizabeth A; Ahlskog, J Eric; Silber, Michael H et al. (2018) Do selective serotonin reuptake inhibitors improve survival in multiple system atrophy? Parkinsonism Relat Disord 48:51-53
McKay, Jake H; Cheshire, William P (2018) First symptoms in multiple system atrophy. Clin Auton Res 28:215-221
Farrell, Maureen C; Brenner, Alexander S; Shibao, Cyndya A (2018) Diagnostic treatment dilemma: baroreflex failure or autoimmune autonomic ganglionopathy? Clin Auton Res 28:589-591
Coon, Elizabeth A; Benarroch, Eduardo E (2018) DNA damage response: Selected review and neurologic implications. Neurology 90:367-376
Palma, Jose-Alberto; Norcliffe-Kaufmann, Lucy; Kaufmann, Horacio (2018) Diagnosis of multiple system atrophy. Auton Neurosci 211:15-25
Walsh, Ryan R; Krismer, Florian; Galpern, Wendy R et al. (2018) Recommendations of the Global Multiple System Atrophy Research Roadmap Meeting. Neurology 90:74-82
Kazachkov, Mikhail; Palma, Jose-Alberto; Norcliffe-Kaufmann, Lucy et al. (2018) Respiratory care in familial dysautonomia: Systematic review and expert consensus recommendations. Respir Med 141:37-46
Aluma, Bat-El Bar; Norcliffe-Kaufmann, Lucy; Sarouk, Ifat et al. (2018) Resting Energy Expenditure in Patients with Familial Dysautonomia: A Preliminary Study. J Pediatr Gastroenterol Nutr :

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