5 Result(s)
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Article
Open AccessInvestigation of non-motor symptoms in patients with amyotrophic lateral sclerosis
[Objective] Few studies have comprehensively investigated the non-motor symptoms of amyotrophic lateral sclerosis (ALS). We aimed to investigate this aspect of ALS. [Methods] We held a nationwide webinar, titl...
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Article
Open AccessEfficacy confirmation study of aceneuramic acid administration for GNE myopathy in Japan
A rare muscle disease, GNE myopathy is caused by mutations in the GNE gene involved in sialic acid biosynthesis. Our recent phase II/III study has indicated that oral administration of aceneuramic acid to patient...
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Article
Open AccessThe updated retrospective questionnaire study of sporadic inclusion body myositis in Japan
Sporadic inclusion body myositis (sIBM) is the most prevalent muscle disease in elderly people, affecting the daily activities. sIBM is progressive with unknown cause and without effective treatment. In 2015, ...
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Article
Open AccessPhase I clinical trial results of aceneuramic acid for GNE myopathy in Japan
GNE myopathy (distal myopathy with rimmed vacuoles) is a rare intractable muscle disease caused by the mutations in GNE gene, with no therapeutic agents at present. The mutations in GNE (UDP-N-acetylglucosamine 2...
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Article
Open AccessMulticenter questionnaire survey for sporadic inclusion body myositis in Japan
Sporadic inclusion body myositis (sIBM) is the most prevalent acquired muscle disease in the elderly. sIBM is an intractable and progressive disease of unknown cause and without effective treatment. The etiolo...
