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

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; Michele Costigan; Emily Roberts; Elizabeth Klingner; Charlie Choi; Dosten Kopenhauszen; M. Quintana; Farah Khandwala; Eric Henninger; William Gahl; Marjan Huizing; Joseph Shrader; Galen Joe; Alessandro Noseda; Giuseppe Testa; Anthony Amato

doi:10.1212/wnl.0000000000217922

Key Points

This research aims to assess whether N-acetylmannosamine can effectively slow muscle strength decline in individuals with GNE myopathy compared to a placebo.
Randomized double-blind placebo-controlled multicenter trial
Participants received either N-acetylmannosamine or placebo
Efficacy evaluated based on muscle strength decline
N-acetylmannosamine group showed a significant slowing of muscle strength decline compared to placebo (exact metrics not provided)

Implication

Randomized trial evaluates muscle strength decline in individuals with GNE myopathy, suggesting potential benefits of treatment.

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.

Discussion

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Cite This Study

Rossignol et al. (2026) studied this question.

synapsesocial.com/papers/6a2cf403faef96ed7f0565a5 https://doi.org/10.1212/wnl.0000000000217922

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

Key Points

Implication

Abstract

Discussion

Cite This Study

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

Key Points

Implication

Abstract

Discussion

Cite This Study

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