A Pivotal, Randomized, Double-Blind, Placebo-Controlled, Multicenter Trial of N-acetylmannosamine (ManNAc) in GNE myopathy: Results from the NN109 MAGiNE Study (LS2.001)

Neurology 2026

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Authors: Francis Rossignol, Christopher Coffey, Merit Cudkowicz, LUMY SAWAKI-ADAMS, Amy Tsou, Richard Barohn, Miriam Freimer, Nuria Carrillo, Timothy Leonard, David Klements, Dixie Ecklund, Maxine Koepp
+15 more Michele Costigan, Emily Roberts, Elizabeth Klingner, Charlie Choi, Dosten Kopenhauszen, Melanie Quintana, Farah Khandwala, Eric Henninger, William Gahl, Marjan Huizing, Joseph Shrader, Galen Joe, Alessandro Noseda, Giuseppe Testa, Anthony Amato
DOI: 10.1212/wnl.0000000000217922
Cited by: 1

Abstract

To evaluate the efficacy of N-acetylmannosamine (ManNAc) administration in individuals with GNE myopathy to slow the progression of muscle strength decline as compared to placebo.

Fields of Study & Subjects

Inflammatory Myopathies and Dermatomyositis Parkinson's Disease and Spinal Disorders Muscle Physiology and Disorders

Publication details

Journal / source
Neurology
Publisher
Ovid Technologies (Wolters Kluwer Health)
Volume
106
Issue
11_Supplement_1
Publication date
9 June 2026
Language
EN