Section 1 of 6
Terms and abbreviations
1 evidence topic · 0 sources
| Abbreviation | Term |
|---|---|
| AHDS | Allan-Herndon-Dudley syndrome |
| AHFS | American Hospital Formulary Service |
| ALT | Alanine aminotransferase |
| AMNOG | Arzneimittelmarktneuordnungsgesetz |
| ANMAT | Administración Nacional de Medicamentos, Alimentos y Tecnología Médica |
| ANOVA | Analysis of variance |
| ASP | Average sales price |
| AST | Aspartate aminotransferase |
| ATC | Anatomical Therapeutic Chemical classification |
| AUC | Area under the concentration-time curve |
| BCRP | Breast cancer resistance protein |
| BMI | Body mass index |
| bpm | Beats per minute |
| BSID-III | Bayley Scales of Infant and Toddler Development, Third Edition |
| BW | Body weight |
| CHMP | Committee for Medicinal Products for Human Use |
| CI | Confidence interval |
| CK | Creatine kinase |
| Cmax | Maximum plasma concentration |
| CNS | Central nervous system |
| COs | Cerebral organoids |
| CPK | Creatine phosphokinase |
| CTCAE | Common Terminology Criteria for Adverse Events |
| CYP | Cytochrome P450 |
| DBP | Diastolic blood pressure |
| DBS | Dried blood spot |
| DD/ID | Developmental delay/intellectual disability |
| DEXA | Dual-energy X-ray absorptiometry |
| DITPA | Diiodothyropropionic acid |
| Dko | Double knockout |
| EAP | Expanded Access Program |
| EEG | Electroencephalography |
| EHR | Electronic health record |
| EMA | European Medicines Agency |
| EMC | Erasmus Medical Center |
| ENT2 | Equilibrative nucleoside transporter 2 |
| EQ-5D-5L | EuroQol 5-Dimension 5-Level questionnaire |
| EU | European Union |
| FDA | Food and Drug Administration |
| FT3 | Free triiodothyronine |
| FT4 | Free thyroxine |
| G | Gastrostomy |
| G-BA | Gemeinsamer Bundesausschuss |
| GI | Gastrointestinal |
| GMFM-88 | Gross Motor Function Measure, 88-item version |
| GRADE | Grading of Recommendations Assessment, Development and Evaluation |
| hERG | Human ether-à-go-go-related gene |
| HPO | Human Phenotype Ontology |
| HR | Hazard ratio |
| HRQoL | Health-related quality of life |
| HTA | Health technology assessment |
| HTN | Hypertension |
| ICD-10 | International Classification of Diseases, Tenth Revision |
| IMP | Investigational medicinal product |
| INN | International nonproprietary name |
| iPSCs | Induced pluripotent stem cells |
| IQR | Interquartile range |
| IQWiG | Institute for Quality and Efficiency in Health Care |
| IR | Immediate release |
| ITT | Intention-to-treat |
| KO | Knockout |
| L/PATH | Likely pathogenic or pathogenic |
| LC-MS | Liquid chromatography-mass spectrometry |
| LC-MS/MS | Liquid chromatography tandem mass spectrometry |
| LCMS/MS | Liquid chromatography-tandem mass spectrometry |
| LLN | Lower limit of normal |
| LoF | Loss of function |
| LT4 | Levothyroxine |
| MATE | Multidrug and toxin extrusion protein |
| MCT8 | Monocarboxylate transporter 8 |
| Mct8KO | Mct8 knockout |
| MedDRA | Medical Dictionary for Regulatory Activities |
| MRI | Magnetic resonance imaging |
| NDA | New Drug Application |
| NDC | National Drug Code |
| NDRI | Norepinephrine-dopamine reuptake inhibitor |
| NG | Nasogastric |
| NGS | Next-generation sequencing |
| OAT | Organic anion transporter |
| OAT7 | Organic anion transporter 7 |
| OATP | Organic anion-transporting polypeptide |
| OCT | Organic cation transporter |
| ODD | Orphan Drug Designation |
| OHSU | Oregon Health & Science University |
| P-gp | P-glycoprotein |
| PAC | Premature atrial contraction |
| PAP | Patient assistance program |
| PDUFA | Prescription Drug User Fee Act |
| PE1 | Primary endpoint 1 |
| PE2 | Primary endpoint 2 |
| PedsQL | Pediatric Quality of Life Inventory |
| PEG | Percutaneous endoscopic gastrostomy |
| PK | Pharmacokinetics |
| PPI | Proton pump inhibitor |
| PPPY | Per patient per year |
| PRV | Priority review voucher |
| PTU | Propylthiouracil |
| PVC | Polyvinyl chloride |
| QTc | QT interval corrected for heart rate |
| ROC | Receiver operating characteristic |
| RPD | Rare Pediatric Disease |
| rT3 | Reverse triiodothyronine |
| RTP | Randomized Treatment Period |
| SAE | Serious adverse event |
| SAP | Statistical Analysis Plan |
| SBP | Systolic blood pressure |
| SD | Standard deviation |
| SDS | Standard deviation score |
| SEP | Somatosensory evoked potential |
| SGB V | Sozialgesetzbuch Fünftes Buch (German Social Code, Book V) |
| SHBG | Sex hormone-binding globulin |
| SHI | Statutory health insurance |
| SI | International System of Units |
| SLC16A2 | Solute carrier family 16 member 2 |
| SLC22A9 | Solute carrier family 22 member 9 |
| SLC29A2 | Solute carrier family 29 member 2 |
| SOC | System organ class |
| T3 | Triiodothyronine |
| T4 | Thyroxine |
| TA3 | 3,3′,5-triiodothyroacetic acid (tiratricol) |
| TBG | Thyroxine-binding globulin |
| TEAE | Treatment-emergent adverse event |
| TH | Thyroid hormone |
| THR | Thyroid hormone receptor |
| Tmax | Time to maximum plasma concentration |
| TOS | Tablet for oral suspension |
| TRIAC | Triiodothyroacetic acid |
| TSH | Thyroid-stimulating hormone |
| TT3 | Total triiodothyronine |
| TT4 | Total thyroxine |
| UGT | Uridine diphosphate glucuronosyltransferase |
| ULN | Upper limit of normal |
| USPTO | United States Patent and Trademark Office |
| VABS-II | Vineland Adaptive Behavior Scales, Second Edition |
| WAC | Wholesale acquisition cost |
| WACC | Weighted average cost of capital |
| WT | Wild type |