Work

Methodology of clinical research in rare diseases: Development of a research program in juvenile neuronal ceroid lipofuscinosis (JNCL) via creation of a patient registry and collaboration with patient advocates

Year: 2013

Type: article

Study design: Observational Study

Source: Contemporary Clinical Trials

DOI: 10.1016/j.cct.2013.04.004

Authors: Elisabeth A. de Blieck, Erika F. Augustine, Frederick J. Marshall, Heather Adams, Jennifer Cialone, Leon Dure, Jennifer M. Kwon, Nicole Newhouse, Katherine Rose, Paul G. Rothberg, Amy Vierhile, Jonathan W. Mink

Institutions: University of Rochester, University of Alabama, University of Alabama at Birmingham

Language: English

Topic: Lysosomal Storage Disorders Research

Subfield: Physiology

Field: Medicine

Domain: Health Sciences

SDG: Good health and well-being

Open Access status: green

Funders: Batten Disease Support and Research Association, University of Rochester, National Institutes of Health, U.S. Food and Drug Administration, National Institute of Neurological Disorders and Stroke

Awards: G1799576560, G2007103789, G2472270058, University of Rochester Child Neurology Academic Development Program, G4277313193, Phase 2 of CellCept for Treatment of Juvenile Neuronal Ceroid Lipofuscinosis, Neurobehavioral Outcomes of Degenerative Neurologic Disease in Childhood

OpenAlex ID: W1985253230

FWCI: 2.072

Cites: 33

Cited by: 27

Related to: 10