GNE Myopathy

13 clinical trials · Condition

Record	Firm	Date
Efficacy Confirmation Study of NPC-09 (NCT04671472)	Nobelpharma	2021-02-08
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) (NCT02736188)	Ultragenyx Pharmaceutical Inc	2016-05-02
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 (NCT02731690)	Ultragenyx Pharmaceutical Inc	2016-04-29
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) (NCT02377921)	Ultragenyx Pharmaceutical Inc	2015-05-20
An Open Label Phase 2 Study of ManNAc in Subjects With GNE Myopathy (NCT02346461)	National Human Genome Research Institute (NHGRI)	2015-02-05
International GNE Myopathy Patient Registry (NCT04009226)	Newcastle University	2014-03-01
GNE-Myopathy Disease Monitoring Program (GNEM-DMP): A Registry and Prospective Observational Natural History Study to Assess GNE Myopathy or Hereditary Inclusion Body Myopathy (HIBM) (NCT01784679)	Ultragenyx Pharmaceutical Inc	2013-04-05
Phase I Clinical Trial of ManNAc in Patients With GNE Myopathy or Hereditary Inclusion Body Myopathy (HIBM) (NCT01634750)	National Human Genome Research Institute (NHGRI)	2012-09-11
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 (NCT01517880)	Ultragenyx Pharmaceutical Inc	2012-05-01

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