New hope for DMD: Long-Term trial of weekly infusions begins
NCT ID NCT07573631
First seen Jun 26, 2026 · Last updated Jul 24, 2026 · Updated 2 times
Summary
This study is for people with Duchenne muscular dystrophy (DMD) who have already taken part in a previous trial of BMN 351. It tests the long-term safety of weekly intravenous infusions of BMN 351 and whether it helps maintain physical function. Only 18 participants will be enrolled, and they must continue taking stable doses of corticosteroids. The goal is to see if BMN 351 can be a safe, long-term treatment option.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- BMN 351 (an antisense oligonucleotide given intravenously)
- What this could lead to
- If successful, this could support BMN 351 as a long-term treatment to slow muscle decline in Duchenne muscular dystrophy.
- What could go wrong
- This is a small, early-phase extension study with only 18 participants, so results may not apply broadly. Long-term safety and effectiveness are still unproven.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Locations
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Fondazione Serena ETS - Centro Clinico NeMO Milano
Milan, Italy, Italy
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Great Ormond Street Hospital NHS Foundation Trust
London, United Kingdom, WC1N 3JH, United Kingdom
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Hospital Viamed Santa Angela De la Cruz
Seville, Spain, 41013, Spain
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Leids Universitair Medisch Centrum
Leiden, Netherlands, 2333 ZA, Netherlands
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UOC Fase I - Fondazione Policlinico Universitario A. Gemelli IRCCS - Universita Cattolica del Sacro Cuore
Rome, Italy, Italy
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Yeditepe University Kosuyolu Hospital
Istanbul, Turkey, Turkey (Türkiye)
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