Gne Myopathy

• CLINICAL_TRIALS trial_result · 2022-05-16
  Clinical 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.

• CLINICAL_TRIALS trial_result · 2019-04-16
  Clinical Trial Update: An Open Label Phase 2 Study of ManNAc in Subjects With GNE Myopathy [Phase 2]

  Phase 2 open-label trial of ManNAc in GNE myopathy completed; results posted in 2019.

• CLINICAL_TRIALS trial_result · 2019-02-19
  Clinical 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.

• CLINICAL_TRIALS trial_result · 2019-02-19
  Clinical Trial Update: A Study to Evaluate the Safety of Aceneuramic Acid Extended Release (Ace-ER; UX001) Tablets in Glucosamine (UDP-N-acetyl)-2-Epimerase (GNE) Myopathy (GNEM) (Also Known as Hereditary Inclusion Body Myopathy [HIBM]) Patients With Severe Ambulatory Impairment [Phase 2]

  The Phase 2 trial of Aceneuramic Acid Extended Release (Ace-ER; UX001) tablets for patients with GNE myopathy was terminated, with results p…

• CLINICAL_TRIALS trial_result · 2018-07-09
  Clinical 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 · 2018-03-13
  Clinical 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…

• CLINICAL_TRIALS trial_result · 2017-10-19
  Clinical 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.

• CLINICAL_TRIALS trial_result · 2016-06-16
  Clinical 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 …