ataluren (Translarna) Report issue

Small molecule Experimental
AACT ChEMBL DrugBank Drug Central KEGG MeSH PubChem repoDB

Active Ingredient History

NOW
  • Now
Ataluren, sold under the brand name Translarna, is a medication for the treatment of Duchenne muscular dystrophy. It was designed by PTC Therapeutics.   Wikipedia

Chemistry

  • SMILES: OC(=O)c1cccc(c1)c2noc(n2)c3ccccc3F
  • InChIKey: OOUGLTULBSNHNF-UHFFFAOYSA-N
  • Mol. Mass: 284.25
  • ALogP: 3.24
  • ChEMBL Molecule:
    ataluren  
More Chemistry

Pharmacology

  • Mechanism of Action:
  • Multi-specific: Missing data
  • Black Box: No
  • Availability: Missing data
  • Delivery Methods: Missing data
  • Pro Drug: No

Alternative names

3-[5-(2-fluorophenyl)-1,2,4-oxadiazol-3-yl]benzoic acid | ataluren | ec-000.2051 | ptc124 | ptc 124 | ptc-124 | translarna

Organizations (13)

Research & Development (13)

Organization Org Type FDA approvals Clinical Trials involvement Org ID Force Sort

Marketing (0)

Organization Org Type FDA approvals Clinical Trials involvement Org ID Force Sort

Indications (19)

Approved Indications (1)


 

Muscular Dystrophies

Experimental Indications - Clinical Trial Phases 1-4 (19)


 

Amino Acid Metabolism, Inborn Errors (Phase 2)

Aniridia (Phase 2)

Colorectal Neoplasms (Phase 1/Phase 2)

Cystic Fibrosis (Phase 4)

Dystrophin (Phase 3)

Endometrial Neoplasms (Phase 1/Phase 2)

Epilepsy (Phase 2)

Genetic Diseases, Inborn (Phase 3)

Genetic Diseases, X-Linked (Phase 3)

Healthy Volunteers (Phase 1)

Hemophilia A (Phase 2)

Hemophilia B (Phase 2)

Muscular Diseases (Phase 3)

Muscular Disorders, Atrophic (Phase 3)

Muscular Dystrophies (Phase 3)

Muscular Dystrophy, Duchenne (Phase 3)

Musculoskeletal Diseases (Phase 3)

Nervous System Diseases (Phase 3)

Neuromuscular Diseases (Phase 3)

Overview

  • Highest Phase: Phase 4
  • # of Trials: 32
  • # of Trial Organizations: 13

Trials by Phase

Trial Phase Start Date Organizations Indications

Feedback

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