Amino Acid Metabolism, Inborn Errors
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Pipeline Programs
2
Companies
1
Clinical Trials
0
Approved Products
Pipeline by Development Stage
Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
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Early DiscoveryClinical DevelopmentMarket
Competitive Landscape
2 companies ranked by most advanced pipeline stage
Trial Timeline
Clinical trial activity over time
2021
2022
2023
2024
2025
2026
PTC TherapeuticsAtaluren
Clinical Trials (1)
Total enrollment: 11 patients across 1 trials
Ataluren for Nonsense Mutation Methylmalonic Acidemia
Start: Jul 2010Est. completion: Nov 201111 patients
Phase 2Terminated
Phase Legend
Preclinical— Lab & animal studies
Phase 1— Safety & dosing
Phase 2— Efficacy testing
Phase 3— Large-scale trials
On Market— Approved & available
Key Insights
2 companies competing in this space
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