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PTC124
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Genetic Markers
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Pathway Enrichment
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Literature
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Targeting translational read-through of premature termination mutations in BMPR2 with PTC124 for pul…
Investigation of PTC124-mediated translational readthrough in a retinal organoid model of AIPL1-asso…
Aminoglycosides, but not PTC124 (Ataluren), rescue nonsense mutations in the leptin receptor and in …
Enhancement of premature stop codon readthrough in the CFTR gene by Ataluren (PTC124) derivatives.
Functional rescue of REP1 following treatment with PTC124 and novel derivative PTC-414 in human chor…
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Clinical Trials
Most Relevant Clinical Trials
PTC124 for the Treatment of Cystic Fibrosis
PTC124 for Cystic Fibrosis
Extension Study of Ataluren (PTC124) in Cystic Fibrosis
Safety and Efficacy Study of PTC124 in Duchenne Muscular Dystrophy
Phase 2a Extension Study of Ataluren (PTC124) in Duchenne Muscular Dystrophy (DMD)
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