Biomarin Pharmaceutical Inc. (BMRN) announced an update on their ongoing clinical study.
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BioMarin Pharmaceutical Inc. (BMRN) is running a follow-on study to track its Duchenne muscular dystrophy drug BMN 351 over the long term. The official title shows it will test weekly infusions in boys whose disease can be treated by skipping exon 51, aiming to confirm lasting safety and real-world gains in physical function.
The treatment is BMN 351, an RNA based drug given by IV once a week. It is designed to help the body make a working form of dystrophin, which could slow muscle damage and improve daily movement in Duchenne patients.
The study is interventional, with no randomization and only one treatment group, so all participants receive BMN 351. It is open label, meaning doctors and families know the treatment, and the main goal is to see how safe and effective the drug remains over time.
The trial opened for invited participants from the earlier 351-201 study and is moving through Phase 2 testing. The latest update was posted on May 12, 2026, signaling active study management and that BioMarin is refining dose decisions as more safety and performance data come in.
For investors, this update supports the view that BioMarin is pushing BMN 351 toward a larger commercial path in Duchenne. Positive long term safety and function data could strengthen BMRN’s rare disease portfolio, influence sentiment in the exon skipping space, and add pressure on rivals working on gene therapies and other RNA based approaches.
The BMN 351 extension trial remains active and updated, with more details available on the ClinicalTrials.gov portal under ID NCT07573631.
To learn more about BMRN’s potential, visit the Biomarin Pharmaceutical Inc. drug pipeline page.