# A Study to Evaluate the Safety and Pharmacokinetics of Ataluren in Participants From ≥6 Months to <2 Years of Age With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)

> **NCT04336826** · PHASE2 · COMPLETED · sponsor: **PTC Therapeutics** · enrollment: 6 (actual)

## Conditions studied

- Nonsene Mutation Duchenne Muscular Dystrophy

## Interventions

- **DRUG:** Ataluren

## Key facts

- **NCT ID:** NCT04336826
- **Lead sponsor:** PTC Therapeutics
- **Sponsor class:** INDUSTRY
- **Phase:** PHASE2
- **Study type:** INTERVENTIONAL
- **Status:** COMPLETED
- **Start date:** 2021-12-29
- **Primary completion:** 2023-08-07
- **Final completion:** 2023-08-07
- **Target enrollment:** 6 (ACTUAL)
- **Last updated:** 2024-04-01


## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT04336826

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT04336826, "A Study to Evaluate the Safety and Pharmacokinetics of Ataluren in Participants From ≥6 Months to <2 Years of Age With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)". Retrieved via AI Analytics 2026-06-02 from https://api.ai-analytics.org/clinical/NCT04336826. Licensed CC0.

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