Hereditary Inclusion Body Myopathy
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CLINICAL_TRIALS trial_result · 2022-05-16Clinical Trial Update: Efficacy Confirmation Study of NPC-09 [Phase 3]
Phase 3 efficacy confirmation study for NPC-09 in GNE myopathy and related distal myopathies has completed.
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CLINICAL_TRIALS trial_result · 2019-02-19Clinical Trial Update: Study to Evaluate the Safety and Efficacy of Aceneuramic Acid Extended-Release (Ace-ER) Tablets in Patients With Glucosamine (UDP-N-acetyl)-2-epimerase Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM) [Phase 3]
Phase 3 trial evaluating Ace-ER in GNEM/HIBM was terminated; results were posted in 2019.
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CLINICAL_TRIALS trial_result · 2018-07-09Clinical Trial Update: Phase 3 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Sialic Acid in Patients With Glucosamine (UDP-N-acetyl)-2-epimerase Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM) [Phase 3]
A Phase 3 randomized, double-blind, placebo-controlled study evaluating sialic acid in GNEM/HIBM (GNE myopathy) has completed with results p…
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CLINICAL_TRIALS trial_result · 2018-03-13Clinical Trial Update: An Open Label Phase 2 Extension Study of Higher Dose Sialic Acid-Extended Release (SA-ER) Tablets and Sialic Acid-Immediate Release (SA-IR) Capsules in Patients With Glucosamine (UDP-N-acetyl)-2-Epimerase (GNE) Myopathy [Phase 2]
Open-label Phase 2 extension study evaluating higher-dose sialic acid formulations (SA-ER and SA-IR) in GNE myopathy; study completed with r…
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CLINICAL_TRIALS trial_result · 2017-10-19Clinical Trial Update: Phase I Clinical Trial of ManNAc in Patients With GNE Myopathy or Hereditary Inclusion Body Myopathy (HIBM) [Phase 1]
Phase I trial of ManNAc (N-acetylmannosamine) in patients with GNE myopathy and hereditary inclusion body myopathy has completed.
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CLINICAL_TRIALS trial_result · 2016-06-16Clinical Trial Update: A Phase 2 Study to Evaluate the Dose and Pharmacodynamic Efficacy of Sialic Acid-Extended Release (SA-ER) Tablets in Patients With GNE Myopathy or Hereditary Inclusion Body Myopathy [Phase 2]
Phase 2 trial evaluating dose and pharmacodynamic efficacy of Sialic Acid-Extended Release (SA-ER) tablets in patients with GNE myopathy or …
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CLINICAL_TRIALS trial_result · 2012-05-21Clinical Trial Update: Safety and Pharmacokinetics of Sialic Acid Tables in Patients With Hereditary Inclusion Body Myopathy (HIBM) [Phase 1]
Phase 1 study assessing safety and pharmacokinetics of Sialic Acid Tables in patients with Hereditary inclusion body myopathy has completed.
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CLINICAL_TRIALS trial_result · 2011-09-08Clinical Trial Update: Pharmacokinetic Study on N-acetylneuraminic Acid [Phase 1]
Phase 1 pharmacokinetic study of N-acetylneuraminic acid conducted by Tohoku University has completed, evaluating pharmacokinetics in the co…