30. Distal myopathy Disease details / Clinical trials / Drug dev / DR info


Clinical trials : 15 Drugs : 17 - (DrugBank : 3) / Drug target gene : 1 - Drug target pathway : 1

Drugs and their primary sponsors and trial info
Aceneuramic Acid Extended-Release   
   Ultragenyx Pharmaceutical Inc
      2016   Phase 2   NCT02731690   Bulgaria;Canada;United States;
Aceneuramic Acid Extended-Release Tablets   
   Ultragenyx Pharmaceutical Inc
      2016   Phase 3   NCT02736188   Bulgaria;Canada;France;Israel;Italy;United Kingdom;United States;
Aceneuramic acid   
   Suzuki Ryoichi
      2021   Phase 3   JPRN-jRCT2021200030   Japan;
Aceneuramic acid extended-release (Ace-ER)   
   Ultragenyx Pharmaceutical Inc
      2015   Phase 3   NCT02377921   Bulgaria;Canada;France;Israel;Italy;United Kingdom;United States;
Acido Sialico - Sialic acid (INN: aceneuramic acid)   
   ULTRAGENYX PHARMACEUTICAL INC.
      2015   Phase 3   EUCTR2014-005432-33-IT   Bulgaria;Canada;France;Israel;Italy;United Kingdom;United States;
Common Name: Sialic Acid   
   ULTRAGENYX PHARMACEUTICAL INC.
      2017   Phase 3   EUCTR2016-000360-42-IT   Bulgaria;Canada;France;Israel;Italy;United Kingdom;
   Ultragenyx Pharmaceutical Inc.
      2017   Phase 3   EUCTR2016-000360-42-BG   Bulgaria;Canada;France;Israel;Italy;United Kingdom;United States;
      2016   Phase 3   EUCTR2016-000360-42-GB   Bulgaria;Canada;France;Israel;Italy;United Kingdom;United States;
Deflazacort   
   Ludwig-Maximilians - University of Munich
      2003   Phase 2/Phase 3   NCT00527228   Germany;
INN: aceneuramic acid   
   Ultragenyx Pharmaceutical Inc.
      2017   Phase 3   EUCTR2016-000360-42-BG   Bulgaria;Canada;France;Israel;Italy;United Kingdom;United States;
      2016   Phase 3   EUCTR2016-000360-42-GB   Bulgaria;Canada;France;Israel;Italy;United Kingdom;United States;
N-acetylneuraminic acid   
   Ultragenyx Pharmaceutical Inc.
      2017   Phase 2   EUCTR2015-004553-41-BG   Bulgaria;Canada;United States;
NPC-09   
   Nobelpharma
      2021   Phase 3   NCT04671472   Japan;
   Tohoku University
      2010   Phase 1   NCT01236898   Japan;
SA   
   Tohoku University Hospital, Department of Neurology
      2017   Phase 2,3   JPRN-UMIN000026354   Japan;
      2016   Phase 2,3   JPRN-UMIN000020683   Japan;
Sialic acid (INN: aceneuramic acid)   
   Ultragenyx Pharmaceutical Inc.
      2017   Phase 2   EUCTR2015-004553-41-BG   Bulgaria;Canada;United States;
      2016   Phase 3   EUCTR2014-005432-33-BG   Bulgaria;Canada;France;Israel;Italy;United Kingdom;United States;
Sialic acid, N-acetylneuraminic acid   
   Ultragenyx Pharmaceutical Inc.
      2017   Phase 3   EUCTR2016-000360-42-BG   Bulgaria;Canada;France;Israel;Italy;United Kingdom;United States;
UX001   
   ULTRAGENYX PHARMACEUTICAL INC.
      2017   Phase 3   EUCTR2016-000360-42-IT   Bulgaria;Canada;France;Israel;Italy;United Kingdom;
      2015   Phase 3   EUCTR2014-005432-33-IT   Bulgaria;Canada;France;Israel;Italy;United Kingdom;United States;
   Ultragenyx Pharmaceutical Inc.
      2017   Phase 3   EUCTR2016-000360-42-BG   Bulgaria;Canada;France;Israel;Italy;United Kingdom;United States;
      2017   Phase 2   EUCTR2015-004553-41-BG   Bulgaria;Canada;United States;
      2016   Phase 3   EUCTR2016-000360-42-GB   Bulgaria;Canada;France;Israel;Italy;United Kingdom;United States;
      2016   Phase 3   EUCTR2014-005432-33-BG   Bulgaria;Canada;France;Israel;Italy;United Kingdom;United States;