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 provides the intellectual capital and the breadth of reach into the Patient Advocate Groups (PAG) community to gain insight into the needs and aspirations of that community from the patients and the caregiver's perspective. 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 PIs of this RDCRC renewal have a long relationship with the PAGs, growing out of the initial one about 20 years ago which focused on Shy-Drager Syndrome (now Multiple System Atrophy, MSA). That relationship has uniquely affected our understanding of the perspective of both patients and caregivers. 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 over $2 million. The administrative structures we have in place should lead to successful biomarker and therapeutic investigations that will improve the health of our patients.

Public Health Relevance

Diseases of the nervous system such as multiple system atrophy strike people in midlife and lead to disability and death over 4-8 years. Our research aims to find the causes of these diseases and use that discovery to find ways to treat them, and hopefully, to eventually prevent them. We believe that the knowledge we gain could also help in understanding other serious diseases such as Alzheimer's disease and Parkinson's disease, which also currently have no cure.

National Institute of Health (NIH)
National Institute of Neurological Disorders and Stroke (NINDS)
Specialized Center--Cooperative Agreements (U54)
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Special Emphasis Panel (ZTR1-CI-8 (01))
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Sieber, Beth-Anne
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Vanderbilt University Medical Center
United States
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Loavenbruck, A; Iturrino, J; Singer, W et al. (2015) Disturbances of gastrointestinal transit and autonomic functions in postural orthostatic tachycardia syndrome. Neurogastroenterol Motil 27:92-8
Palma, Jose-Alberto; Kaufmann, Horacio (2015) Novel therapeutic approaches in multiple system atrophy. Clin Auton Res 25:37-45
Piccione, Ezequiel A; Sletten, David M; Staff, Nathan P et al. (2015) Autonomic system and amyotrophic lateral sclerosis. Muscle Nerve 51:676-9
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Palma, Jose-Alberto; Kaufmann, Horacio (2014) Autonomic disorders predicting Parkinson's disease. Parkinsonism Relat Disord 20 Suppl 1:S94-8

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