Section 1 of 6
Terms and abbreviations
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| Abbreviation | Term |
|---|---|
| AAN | American Academy of Neurology |
| AHFS | American Hospital Formulary Service |
| ASGCT | American Society of Gene and Cell Therapy |
| ASO | Antisense oligonucleotide |
| BLA | Biologics License Application |
| BMD | Becker muscular dystrophy |
| CAP-1002 | Development code for deramiocel |
| CDCs | Cardiosphere-derived cells |
| CI | Confidence interval |
| CK | Creatine kinase |
| cMRI | Cardiac magnetic resonance imaging |
| DMD | Duchenne muscular dystrophy |
| DMD-QoL | Duchenne Muscular Dystrophy Quality of Life questionnaire |
| EC | External comparator |
| ECP | External comparator population |
| FDA | U.S. Food and Drug Administration |
| GMP | Good manufacturing practice |
| hCDC-EVs | Human cardiosphere-derived cell extracellular vesicles |
| HDAC | Histone deacetylase |
| HRQoL | Health-related quality of life |
| HUI-2 | Health Utilities Index Mark 2 |
| ICC | Intraclass correlation coefficient |
| IQR | Interquartile range |
| ITT | Intention to treat |
| IV | Intravenous |
| LGMD | Limb-girdle muscular dystrophy |
| LVEF | Left ventricular ejection fraction |
| mCDCs | Mouse cardiosphere-derived cells |
| MD | Muscular dystrophy |
| MDA | Muscular Dystrophy Association |
| mdx | Dystrophin-deficient mouse model |
| MMRM | Mixed model for repeated measures |
| NSAA | North Star Ambulatory Assessment |
| OLE | Open-label extension |
| PDUFA | Prescription Drug User Fee Act |
| PPMD | Parent Project Muscular Dystrophy |
| PUL | Performance of the Upper Limb |
| RMAT | Regenerative Medicine Advanced Therapy |
| SAP | Statistical analysis plan |
| SD | Standard deviation |
| SLRs | Systematic literature reviews |
| USD | United States dollars |