Experimental drug shows promise in slowing duchenne muscle loss

NCT ID NCT04433234

Ongoing Disease control Sponsor: Daiichi Sankyo Co., Ltd. Source: ClinicalTrials.gov ↗

First seen Jun 26, 2026 · Last updated Jun 26, 2026

Summary

This study tests the long-term safety and effectiveness of an experimental drug called DS-5141b (Renadirsen) in 8 people with Duchenne muscular dystrophy who have already completed a previous study. Participants receive a weekly injection under the skin. Researchers will monitor side effects and measure changes in walking, muscle strength, and breathing over time.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: DS-5141b (Renadirsen), given as a weekly injection under the skin
What this could lead to: If successful, this could point toward a treatment that slows muscle decline and helps maintain walking ability in Duchenne muscular dystrophy.
What could go wrong: This is a very small, early-phase study with only 8 participants, so results may not apply to all patients. Long-term safety and effectiveness are still uncertain.

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Conditions

The condition(s) this trial relates to.

Duchenne muscular dystrophy

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

DUCHENNE MUSCULAR DYSTROPHY

Contacts and locations

Locations

Kobe University Hospital

Hyōgo, 650-0017, Japan
National Center of Neurology and Psychiatry

Tokyo, Kodaira-Shi, 187-8551, Japan

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