New hope for duchenne? experimental drug BMN 351 enters human testing

NCT ID NCT06280209

Ongoing Disease control Sponsor: BioMarin Pharmaceutical Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This early-stage trial is testing a drug called BMN 351 in 18 boys aged 4 to 10 with Duchenne muscular dystrophy who have a specific genetic change. The drug is designed to help the body produce a shorter but still useful version of the muscle protein dystrophin. The main goal is to check the drug's safety and how the body processes it, not yet to prove it works.

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 designed to skip exon 51 in the dystrophin gene)
What this could lead to: If successful, this could lead to a treatment that helps boys with Duchenne muscular dystrophy produce a shorter but functional version of the dystrophin protein, potentially slowing muscle decline.
What could go wrong: This is an early phase 1/2 trial with only 18 participants, so safety and dosing are still being established. It is not yet known if BMN 351 will meaningfully improve muscle function or quality of life.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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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

Fondazione Serena ETS - Centro Clinico NeMO Milano

Milan, Italy
Great Ormond Street Hospital NHS Foundation Trust

London, WC1N 3JH, United Kingdom
Hospital Sant Joan de Deu

Barcelona, 08950, Spain
Hospital Viamed Santa Angela De la Cruz

Seville, 41013, Spain
Leids Universitair Medisch Centrum

Leiden, 2333 ZA, Netherlands
UOC Fase I - Fondazione Policlinico Universitario A. Gemelli IRCCS - Universita Cattolica del Sacro Cuore

Rome, Italy
Yeditepe University Kosuyolu Hospital

Istanbul, Turkey (Türkiye)

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