Multi-Center Study of ManNAc for GNE Myopathy (MAGiNE)
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|ClinicalTrials.gov Identifier: NCT04231266|
Recruitment Status : Not yet recruiting
First Posted : January 18, 2020
Last Update Posted : February 6, 2020
William A. Gahl, MD, PhD
Brigham and Women's Hospital
National Human Genome Research Institute (NHGRI)
National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
National Institute of Neurological Disorders and Stroke (NINDS)
Leadiant Biosciences, Inc.
Information provided by (Responsible Party):
William A. Gahl, MD, PhD, National Human Genome Research Institute (NHGRI)
No Study Results Posted on ClinicalTrials.gov for this Study
|Recruitment Status :||Not yet recruiting|
|Estimated Primary Completion Date :||January 31, 2023|
|Estimated Study Completion Date :||January 31, 2023|
Quintana M, Shrader J, Slota C, Joe G, McKew JC, Fitzgerald M, Gahl WA, Berry S, Carrillo N. Bayesian model of disease progression in GNE myopathy. Stat Med. 2019 Apr 15;38(8):1459-1474. doi: 10.1002/sim.8050. Epub 2018 Dec 3.
Carrillo N, Malicdan MC, Huizing M. GNE Myopathy: Etiology, Diagnosis, and Therapeutic Challenges. Neurotherapeutics. 2018 Oct;15(4):900-914. doi: 10.1007/s13311-018-0671-y. Review.
Xu X, Wang AQ, Latham LL, Celeste F, Ciccone C, Malicdan MC, Goldspiel B, Terse P, Cradock J, Yang N, Yorke S, McKew JC, Gahl WA, Huizing M, Carrillo N. Safety, pharmacokinetics and sialic acid production after oral administration of N-acetylmannosamine (ManNAc) to subjects with GNE myopathy. Mol Genet Metab. 2017 Sep;122(1-2):126-134. doi: 10.1016/j.ymgme.2017.04.010. Epub 2017 Apr 26.