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Rare Disease Research, Llc

Clinical trials sponsored by Rare Disease Research, Llc, explained in plain language.

DMD drug safety trial ends early with just 2 participants

Disease control Terminated

This study looked at the safety of a drug called golodirsen in boys with Duchenne muscular dystrophy who can no longer walk. The drug is given as a weekly IV infusion. The study was stopped early and only included 2 participants, so results are very limited.

Phase: PHASE4 • Sponsor: Rare Disease Research, LLC • Aim: Disease control

Last updated May 26, 2026 07:03 UTC