Phase II/III Study of Aceneuramic Acid Administration for GNE Myopathy in Japan.
Sign Up to like & getrecommendations! Published in 2023 at "Journal of neuromuscular diseases"
DOI: 10.3233/jnd-230029
Abstract: BACKGROUND GNE myopathy is an ultra-rare muscle disease characterized by a reduction in the synthesis of sialic acid derived from pathogenic variants in the GNE gene. No treatment has been established so far. OBJECTIVE We… read more here.
Keywords: gne myopathy; administration; group; aceneuramic acid ... See more keywords