246. Methylmalonic acidemia Disease details / Clinical trials / Drug dev / DR info


Clinical trials : 23 Drugs : 31 - (DrugBank : 8) / Drug target genes : 17 - Drug target pathways : 23

Drugs and their primary sponsors and trial info
291844   
   PTC Therapeutics, Inc.
      2010   Phase 2   EUCTR2009-016654-41-GB   Belgium;France;Italy;United Kingdom;
      2010   -   EUCTR2009-016654-41-FR   France;Italy;United Kingdom;
Ataluren   
   PTC Therapeutics
      2010   Phase 2   NCT01141075   Belgium;France;Germany;Italy;Switzerland;United Kingdom;
   PTC Therapeutics, Inc.
      2010   Phase 2   EUCTR2009-016654-41-GB   Belgium;France;Italy;United Kingdom;
      2010   Phase 2   EUCTR2009-016654-41-BE   Belgium;France;Italy;Switzerland;United Kingdom;
      2010   -   EUCTR2009-016654-41-FR   France;Italy;United Kingdom;
BBP-671   
   CoA Therapeutics, Inc., a BridgeBio company
      2021   Phase 1   NCT04836494   United States;
Bucladesine   
   Ito Tetsuya
      2021   Phase 1-2   JPRN-jRCT2041210065   Japan;
Carbaglu   
   Mendel Tuchman
      2012   Phase 2   NCT01599286   United States;
Carglumic Acid (Carbaglu®)   
   National Taiwan University Hospital
      2019   -   NCT04284917   Taiwan;
Compound 1a   
   PTC Therapeutics, Inc.
      2010   Phase 2   EUCTR2009-016654-41-GB   Belgium;France;Italy;United Kingdom;
      2010   -   EUCTR2009-016654-41-FR   France;Italy;United Kingdom;
HLB-001   
   LogicBio Therapeutics, Inc.
      2021   Phase 1/Phase 2   NCT04581785   Saudi Arabia;United States;
HST5040   
   HemoShear Therapeutics
      2021   Phase 2   NCT04732429   United States;
MRNA-3704   
   ModernaTX, Inc.
      2019   Phase 1;Phase 2   EUCTR2019-001061-32-GB   United Kingdom;United States;
      2019   Phase 1/Phase 2   NCT03810690   United States;
MRNA-3705   
   ModernaTX, Inc.
      2021   Phase 1/Phase 2   NCT04899310   Canada;United Kingdom;
PTC-0161480   
   PTC Therapeutics, Inc.
      2010   Phase 2   EUCTR2009-016654-41-GB   Belgium;France;Italy;United Kingdom;
      2010   -   EUCTR2009-016654-41-FR   France;Italy;United Kingdom;
PTC-124   
   PTC Therapeutics, Inc.
      2010   Phase 2   EUCTR2009-016654-41-GB   Belgium;France;Italy;United Kingdom;
      2010   -   EUCTR2009-016654-41-FR   France;Italy;United Kingdom;
PTC-C124   
   PTC Therapeutics, Inc.
      2010   Phase 2   EUCTR2009-016654-41-GB   Belgium;France;Italy;United Kingdom;
      2010   -   EUCTR2009-016654-41-FR   France;Italy;United Kingdom;
PTC124   
   PTC THERAPEUTICS, INC.
      2010   -   EUCTR2009-016654-41-IT   France;Italy;United Kingdom;
   PTC Therapeutics, Inc.
      2010   Phase 2   EUCTR2009-016654-41-GB   Belgium;France;Italy;United Kingdom;
      2010   Phase 2   EUCTR2009-016654-41-BE   Belgium;France;Italy;Switzerland;United Kingdom;
      2010   -   EUCTR2009-016654-41-FR   France;Italy;United Kingdom;
RPS 2505   
   PTC Therapeutics, Inc.
      2010   Phase 2   EUCTR2009-016654-41-GB   Belgium;France;Italy;United Kingdom;
      2010   -   EUCTR2009-016654-41-FR   France;Italy;United Kingdom;