A study to evaluate the long-term safety and effectiveness of BMN 351 in patients with Duchenne muscular dystrophy amenable to exon 51 skipping.

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What is this study about?

This study focuses on Duchenne Muscular Dystrophy, a condition that causes progressive muscle weakness and loss of muscle function. The investigation aims to evaluate the long-term safety and effectiveness of a medication known as BMN 351. This drug is a type of exon skipping therapy, which is a method used to help the body produce a more functional version of a protein that is missing or broken due to genetic errors. Specifically, it is designed for individuals whose genetic makeup allows for this specific treatment approach.

The treatment involves intravenous infusion, meaning the medication is delivered directly into a vein through a tube. During this study, participants receive these infusions on a weekly basis. The process is designed to monitor how the body reacts to the medication over an extended period of time to ensure it can be used safely over the long term.

Who Can Join the Study?

Participants must have previously finished the 351-201 or 351-203 studies without stopping the investigational medicinal product, which is the experimental medicine being tested, or leaving the study early.
Participants must currently be taking oral corticosteroids, which are a type of medicine used to reduce inflammation, and must stay on a steady, unchanging dose throughout the study, unless the dose needs to change because of changes in the person’s weight.
Sexually active males must be willing to use contraception, which is a method used to prevent pregnancy, during the study and for 90 days after taking the last dose.
The study is specifically for male patients.

Who Cannot Join the Study?

You cannot participate if you have a known coagulation disorder, which is a condition that affects your body’s ability to form blood clots to stop bleeding.
The study is only for males, so females are not eligible.
The study is designed for a specific age range, so if you are outside the required age group, you cannot join.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country	Status
Fondazione Policlinico Universitario Agostino Gemelli IRCCS	Rome	Italy

Other Sites

Site Name	City	Country	Status
Cquven Cxokxam Nzyx	Milan	Italy
Lplcj Upumfslbmglx Mzdwngu Ccaudog (qogeu	Leiden	The Netherlands

Trial status

Country	Status	Recruitment Start
Italy	Not yet recruiting	20.04.2026
Spain	Not yet recruiting	20.04.2026
The Netherlands	Not yet recruiting	20.04.2026

Trial locations

BMN 351 is a therapy given through an intravenous infusion (a liquid delivered directly into a vein) that is designed to help the body skip a specific part of a gene called exon 51. This process aims to help the body produce a more functional version of a protein that is missing in people with Duchenne Muscular Dystrophy.

Investigated diseases:

Duchenne muscular dystrophy

Duchenne Muscular Dystrophy – This is a genetic condition that primarily affects the muscles. It is caused by a lack of a protein called dystrophin, which helps keep muscle cells intact. Over time, the muscles become progressively weaker. This weakness typically starts in the muscles closer to the center of the body, such as the hips and legs. As the condition continues, the muscle tissue is gradually replaced by fat and fibrous tissue.

Trial ID:

2024-511656-41-00

Protocol code:

351-202

Trial Phase:

Therapeutic exploratory (Phase II)

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A study to evaluate the long-term safety and effectiveness of BMN 351 in patients with Duchenne muscular dystrophy amenable to exon 51 skipping.

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?