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Oculopharyngeal Muscular Dystrophy - 1 Studies Found
| Status | Study |
| RECRUITING |
Study Name: A Study to Evaluate the Safety and Clinical Activity of Intramuscular Doses of BB-301 Administered to Subjects With Oculopharyngeal Muscular Dystrophy With Dysphagia Condition: Oculopharyngeal Muscular Dystrophy Date: 2024-12-08 Interventions: BB-301 is composed of an AAV9 capsid, AAV9PL, which delivers the gene of interest, comprised of a recombinant genome encoding a single RNA transcri |