Section 1 of 6
Terms and abbreviations
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| Abbreviation | Term |
|---|---|
| 10MWR | 10-meter walk/run |
| 4SC | Four-stair climb |
| 6MWD | Six-minute walk distance |
| 6MWT | Six-minute walk test |
| ACE | Angiotensin-converting enzyme |
| AHFS | American Hospital Formulary Service |
| ARB | Angiotensin receptor blocker |
| ASO | Antisense oligonucleotide |
| BLA | Biologics License Application |
| CI | Confidence interval |
| CNS | Central nervous system |
| DMD | Duchenne muscular dystrophy |
| DMD-QoL | Duchenne Muscular Dystrophy Quality of Life measure |
| DYNE-251 | Development code for zeleciment rostudirsen |
| EMA | European Medicines Agency |
| EMQN | European Molecular Genetics Quality Network |
| Fab | Antigen-binding fragment |
| FDA | U.S. Food and Drug Administration |
| FORCE | Dyne Therapeutics antibody-oligonucleotide delivery platform |
| FVC | Forced vital capacity |
| GC | Glucocorticoid |
| HCRU | Healthcare-resource utilization |
| HDAC | Histone deacetylase |
| HR | Hazard ratio |
| HS | Health state |
| HTA | Health technology assessment |
| HUI-2 | Health Utilities Index Mark 2 |
| ICER | Institute for Clinical and Economic Review |
| IV | Intravenous |
| LoA | Loss of ambulation |
| LTE | Long-term extension |
| MAD | Multiple ascending dose |
| MCID | Minimal clinically important difference |
| NSAA | North Star Ambulatory Assessment |
| OLE | Open-label extension |
| PDUFA | Prescription Drug User Fee Act |
| PMO | Phosphorodiamidate morpholino oligomer |
| PPMD | Parent Project Muscular Dystrophy |
| Q1 | First calendar quarter |
| Q4W | Every four weeks |
| REC | Registrational expansion cohort |
| RFF | Rise from floor |
| SD | Standard deviation |
| siRNA | Small interfering RNA |
| SV95C | Stride velocity 95th centile |
| TEAE | Treatment-emergent adverse event |
| TfR1 | Transferrin receptor 1 |
| TTE | Target trial emulation |
| TTR | Time to rise |
| UK | United Kingdom |
| US | United States |
| z-rostudirsen | Zeleciment rostudirsen |