Oculopharyngeal Muscular Dystrophy - 1 Studies Found
| RECRUITING |
: A Study to Evaluate the Safety and Clinical Activity of Intramuscular Doses of BB-301 Administered to Subjects With Oculopharyngeal Muscular Dystrophy With Dysphagia : Oculopharyngeal Muscular Dystrophy : 2024-11-21 : 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 |