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SKY-0515

Huntington's disease

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Section 1 of 6

Terms and abbreviations

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AbbreviationTerm
AANAmerican Academy of Neurology
AEAdverse event
AHFSAmerican Hospital Formulary Service
AMCPAcademy of Managed Care Pharmacy
ANCOVAAnalysis of covariance
ANZCTRAustralian New Zealand Clinical Trials Registry
AOHDAdult-onset Huntington's disease
ASAdvanced stage
ASOAntisense oligonucleotide
BIABudget impact analysis
BLABiologics License Application
C-SSRSColumbia-Suicide Severity Rating Scale
CAGCytosine-adenine-guanine trinucleotide repeat
CAPCAG-age product
CEPACComparative Effectiveness Public Advisory Council
CHMPCommittee for Medicinal Products for Human Use
CIConfidence interval
CNCaudate nucleus
COECenter of Excellence
CRDCentre for Rare Disorders (Oslo University Hospital)
CSFCerebrospinal fluid
CTAClinical Trial Application
cUHDRSComposite Unified Huntington's Disease Rating Scale
DCLDiagnostic confidence level
DGNGerman Neurological Society (Deutsche Gesellschaft für Neurologie)
DMTDisease-modifying treatment
DSMBData Safety Monitoring Board
ECGElectrocardiogram
EHDNEuropean Huntington's Disease Network
EMAEuropean Medicines Agency
EQ-5DEuroQol 5-dimension questionnaire
ESEarly stage
FDAUS Food and Drug Administration
GeM-HDGenetic Modifiers of Huntington's Disease Consortium
GINAGenetic Information Nondiscrimination Act
GWAGenome-wide association
GWASGenome-wide association study
H-QoL-IHuntington Quality of Life Instrument
HCDMHealth care decision-maker
HDHuntington's disease
HD-ISSHuntington's Disease Integrated Staging System
HDBOIHuntington's Disease Burden of Illness study
HDQLIFEHuntington Disease Health-related Quality of Life measurement system
HDSAHuntington's Disease Society of America
HEHighly expanded (CAG repeat subgroup)
HRHazard ratio
HRECHuman Research Ethics Committee
HRQoLHealth-related quality of life
HRUHealthcare resource utilization
HSGHuntington Study Group
HTTHuntingtin gene or protein
ICERInstitute for Clinical and Economic Review; incremental cost-effectiveness ratio
INDInvestigational New Drug application
IQRInterquartile range
ISIndependence Scale
ISPORProfessional Society for Health Economics and Outcomes Research (formerly International Society for Pharmacoeconomics and Outcomes Research)
JHDJuvenile Huntington's disease
JoHDJuvenile-onset Huntington's disease
KMKaplan-Meier
Kp,uuUnbound CSF-to-unbound plasma concentration ratio
LELow expansion (CAG repeat subgroup)
LoHDLate-onset Huntington's disease
LSLeast squares
LTCLong-term care
MAAMarketing Authorisation Application
MADMultiple ascending dose
MDSInternational Parkinson and Movement Disorder Society; movement disorders specialist
MDTMultidisciplinary team
mHTTMutant huntingtin protein
MMRDNA mismatch repair
MRIMagnetic resonance imaging
MSMid stage
MSH3MutS homolog 3
NCDRNorwegian Cause of Death Registry
NfLNeurofilament light chain
NHSNational Health Service
OLEOpen-label extension
PFDPrefunctional decline
PKPharmacokinetics
PMPMPer member per month
PMS1PMS1 homolog 1, mismatch repair system component (postmeiotic segregation increased 1)
PPPMPer patient per month
PREDICT-HDNeurobiological Predictors of Huntington's Disease study
PTMPMPer treated member per month
PwHDPeople with Huntington's disease
PYPerson-years
QALYQuality-adjusted life year
QDOnce daily
RCTRandomized controlled trial
SADSingle ascending dose
SAESerious adverse event
SDStandard deviation
SDMTSymbol Digit Modalities Test
SEStandard error
SF-6DShort-Form 6-Dimension
SF1, SF2Shoulson and Fahn stage 1, stage 2
SNFSkilled nursing facility
SNPSingle nucleotide polymorphism
SNRISerotonin-norepinephrine reuptake inhibitor
SoCStandard of care
SSRISelective serotonin reuptake inhibitor
SWRTStroop Word Reading Test
TEAETreatment-emergent adverse event
TFCTotal Functional Capacity
TGATherapeutic Goods Administration (Australia)
tHTTTotal huntingtin protein
TMSTotal Motor Score
TRACK-HDTRACK-HD observational study
TTOTime trade-off
UHDRSUnified Huntington's Disease Rating Scale
UKUnited Kingdom
USUnited States
VMAT2Vesicular monoamine transporter 2
vMRIVolumetric magnetic resonance imaging
WTWild-type
WTPWillingness to pay