Article

Efficacy Confirmation Study of Aceneuramic Acid Administration for GNE Myopathy in Japan

2023-05-11

Abstract excerpt

<title>Abstract</title> <p><bold>Background</bold> A rare muscle disease, GNE myopathy is caused by mutations in the <italic>GNE</italic> gene involved in the sialic acid biosynthesis. Our recent phase II/III study has indicated that oral administration of aceneuramic acid to patients would slow disease progression. <bold>Methods</bold> We conducted a phase III, randomized, placebo-controlled, double-blind, paral...

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Literature Corpus work: 666cb41e-f0a0-5caf-a71b-1d3d5d030a3d
DOI: 10.21203/rs.3.rs-2874819/v1

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