# An Open-Label Study of Golodirsen in Non-Ambulant Patients With Duchenne Muscular Dystrophy

> **NCT04708314** · PHASE4 · TERMINATED · sponsor: **Rare Disease Research, LLC** · enrollment: 2 (actual)

## Conditions studied

- Duchenne Muscular Dystrophy

## Interventions

- **DRUG:** Golodirsen 50 MG/1 ML Intravenous Solution [VYONDYS 53]

## Key facts

- **NCT ID:** NCT04708314
- **Lead sponsor:** Rare Disease Research, LLC
- **Sponsor class:** OTHER
- **Phase:** PHASE4
- **Study type:** INTERVENTIONAL
- **Status:** TERMINATED
- **Start date:** 2020-10-31
- **Primary completion:** 2021-05-13
- **Final completion:** 2021-05-13
- **Target enrollment:** 2 (ACTUAL)
- **Why stopped:** The Sponsor has decided to focus their resources on other areas of therapy.
- **Last updated:** 2026-04-03

## Collaborators

- [object Object]

## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT04708314

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT04708314, "An Open-Label Study of Golodirsen in Non-Ambulant Patients With Duchenne Muscular Dystrophy". Retrieved via AI Analytics 2026-06-24 from https://api.ai-analytics.org/clinical/NCT04708314. Licensed CC0.

---

*[Clinical trials dataset](/datasets/clinical-trials) · CC0 1.0*