Work

Batten disease: an expert update on agents in preclinical and clinical trials

Year: 2020

Type: editorial

Abstract: The Neuronal Ceroid Lipofuscinoses (NCLs; Batten disease) are a group of rare inherited fatal diseases that are characterized by the buildup of autofluorescent lipopigments in lysosomes [1]. These ...

Source: Expert Opinion on Investigational Drugs

DOI: 10.1080/13543784.2020.1837110

Authors: Margaux C. Masten, Jonathan W. Mink, Erika F. Augustine

Institutions: University of Rochester Medicine, University of Rochester

Language: English

Topic: Lysosomal Storage Disorders Research

Subfield: Physiology (medical)

Field: Medicine

Domain: Health Sciences

SDG: Good health and well-being

Open Access status: green

Funders: National Institute of Neurological Disorders and Stroke, Eunice Kennedy Shriver National Institute of Child Health and Human Development

Awards: University of Rochester Intellectual and Developmental Disabilities Research Center, Meaningful Outcomes and Multi-Site Readiness for Clinical Trials in Juvenile Neuronal Ceroid Lipofuscinosis

OpenAlex ID: W3095146947

Cites: 21

Cited by: 14

Related to: 10