| name: | DelandistrogeneMoxeparvovec | |
| ATC code: | M09AX15 | route: | intravenous | 
| compartments: | 1 | |
| dosage: | 1.33 | mg | 
| volume of distribution: | 1 | L | 
| clearance: | 0 | |
| other parameters in model implementation | ||
Delandistrogene moxeparvovec is a gene therapy consisting of a recombinant adeno-associated virus vector (AAVrh74) encoding a micro-dystrophin gene, designed as a disease-modifying treatment for Duchenne Muscular Dystrophy (DMD) in pediatric patients. As of 2024, it is approved by the US FDA for certain ambulatory children with DMD aged 4-5 years.
No published pharmacokinetic data describing PK parameters (such as volume of distribution, clearance, or bioavailability) are available in peer-reviewed literature or regulatory documents for delandistrogene moxeparvovec (SRP-9001). The product is an intravenous gene therapy, and typical small-molecule PK concepts are not directly applicable.