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January 26, 2019Open Access

A phase 3 randomized study evaluating sialic acid extended-release for GNE myopathy

HLHanns LochmüllerSemmelweis University ABAnthony BéhinSorbonne Université YCYoseph CaracoHadassah Medical Center

Key Points

Key points are not available for this paper at this time.

Abstract

This study provides Class I evidence that for patients with GNE myopathy, Ace-ER does not improve muscle strength compared to placebo.

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Cite This Study

Lochmüller et al. (Sat,) studied this question.

synapsesocial.com/papers/69ceeea6c4d4abef46a4a12a https://doi.org/https://doi.org/10.1212/wnl.0000000000006932

A phase 3 randomized study evaluating sialic acid extended-release for GNE myopathy | Synapse

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4Construction and validation of a quality of life questionnaire for neuromuscular disease (INQoL)2007 · 167 citations
5A Japanese patient with distal myopathy with rimmed vacuoles: missense mutations in the epimerase domain of the UDP-N-acetylglucosamine 2-epimerase/N-acetylmannosamine kinase (GNE) gene accompanied by hyposialylation of skeletal muscle glycoproteins2003 · 63 citations