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Compound 1a

3 trials

Sponsors

PTC Therapeutics, Inc, PTC Therapeutics, Inc.

Conditions

Nonsense Mutation Methylmalonic Acidemia MedDRA version: 12.1 Level: LLT Classification code 10064180 Term: Blood methylmalonic acid increasedNonsense-Mutation-Mediated Cystic Fibrosis MedDRA version: 9.1 Level: LLT Classification code 10011762 Term: Cystic fibrosisNonsense-Mutation-Mediated Hemophilia A and B MedDRA version: 12.1 Level: LLT Classification code 10060613 Term: Hemophilia A (Factor VIII) MedDRA version: 12.1 Level: LLT Classification code 10060614 Term: Hemophilia B (Factor IX)

Phase 2

A Phase 2 Study of Ataluren (PTC124) as an Oral Treatment for Nonsense Mutation Methylmalonic Acidemia
Active, not recruitingEUCTR2009-016654-41-FR
PTC Therapeutics, Inc.Nonsense Mutation Methylmalonic Acidemia MedDRA version: 12.1 Level: LLT Classification code 10064180 Term: Blood methylmalonic acid increased
Start: 2010-03-02Target: 18Updated: 2022-04-04
A Phase 2a Study of Ataluren (PTC124) as an Oral Treatment for Nonsense-Mutation-Mediated Hemophilia A and B
Active, not recruitingEUCTR2010-020224-22-FR
PTC Therapeutics, IncNonsense-Mutation-Mediated Hemophilia A and B MedDRA version: 12.1 Level: LLT Classification code 10060613 Term: Hemophilia A (Factor VIII) MedDRA version: 12.1 Level: LLT Classification code 10060614 Term: Hemophilia B (Factor IX)
Start: 2010-08-26Target: 30Updated: 2022-04-04

Unknown Phase

A Phase 3 Efficacy and Safety Study of PTC124 as an Oral Treatment for Nonsense-Mutation-Mediated Cystic Fibrosis
Active, not recruitingEUCTR2008-003924-52-SE
PTC Therapeutics, IncNonsense-Mutation-Mediated Cystic Fibrosis MedDRA version: 9.1 Level: LLT Classification code 10011762 Term: Cystic fibrosis
Start: 2009-04-08Target: 208Updated: 2012-11-19