Huntington Disease
Watchlist
Retrieved
2022-04-26
Source
Trials
—
Genes
HTT,
CNR1,
OGG1,
GDNF,
SLC2A3,
MAOA,
AIFM1,
SLC29A1,
FAAH,
RCAN1,
DIABLO,
PPARGC1A,
PPP1R1B,
MAOB,
IP6K2,
GAP43,
NRSN1,
GAL,
NFE2L2,
RELN,
CNTF,
ZDHHC17,
CREB1,
NPY,
LEP,
ITPR1,
MMP9,
PPARD,
ZDHHC13,
CNR2
HTT,
CNR1,
OGG1,
GDNF,
SLC2A3,
MAOA,
AIFM1,
SLC29A1,
FAAH,
RCAN1,
DIABLO,
PPARGC1A,
PPP1R1B,
MAOB,
IP6K2,
GAP43,
NRSN1,
GAL,
NFE2L2,
RELN,
CNTF,
ZDHHC17,
CREB1,
NPY,
LEP,
ITPR1,
MMP9,
PPARD,
ZDHHC13,
CNR2,
BCL2,
DRD1,
DUSP1,
PTGS2,
GSR,
ABAT,
BAX,
HAAO,
QPRT,
NOS3,
E2F1,
ND3,
MBP,
CAT,
ADD1,
FAN1,
RNF4,
ADGB,
SH3BP2,
SIRT1,
REST,
RGS12,
MSANTD1,
TP53,
ATXN1,
PDE10A,
ATXN3,
CASP6,
GABPA,
IGF1,
SNCA,
SOD1,
JPH3,
EPO,
TGM2,
SIGMAR1,
GRK4,
TBP,
IL6,
GRIN2B,
HTT-AS,
APOE,
ALB,
BDNF,
HAP1,
AR,
CRP,
DRD2,
DNM1L,
FXN,
GFAP,
SLC6A4,
GRIK2,
CREBBP,
HSF1,
CTNNB1,
GAPDH,
SIRT2,
ACE,
TNF,
DNAJB6,
EIF4E,
ZNF141,
PENK,
OPN1LW,
PTH,
PAG1,
IGFALS,
CDK5,
ATM,
CSF2,
MAPK1,
PCBP4,
MAPT,
SLC1A2,
NEFL,
SMUG1,
C9orf72,
HSPA4,
IFNL3,
HIP1,
NEAT1,
FGF23,
HCRT,
SOST,
GRM5,
IL1B,
LAMC2,
HDAC4,
SETDB1,
NTRK2,
IL10,
TAC1,
CASP3,
PPARG,
KMO,
CALB1,
ATXN2,
PPP1R2C,
GRIN2A,
F8A1,
DAPK2,
LINC01672,
TLR4,
HDAC9,
F8A3,
ADORA2A,
F8A2,
DENR,
HSP90AA1,
MSH3,
AKT1,
SQSTM1,
VEGFA,
MSH2,
PON1,
PTX3,
CRMP1,
SIRT3,
UBB,
IL12B,
UTRN,
H2AX,
HAMP,
ACTB,
ATF6,
CXCL8,
IL12A,
GCG,
FOXO3,
TAL1,
SLC18A2,
ANGPT1,
LY6E,
IDO1,
FUS,
SCLY,
KRT7,
CCL5,
MLH1,
ATXN7,
HIPK3,
HDAC1,
OPTN,
SST,
PPP1R11,
TSHZ1,
SRSF6,
AGER,
APP,
STAT3,
GPT,
ACBD3,
CCL2,
BCL11B,
FOXO1,
CDKL1,
TCERG1,
UCHL1,
BRCA1,
DLG4,
CDK20,
PDE4A,
TARDBP,
ABCB1,
KCNH4,
PIK3CA,
PIK3CD,
PIK3CG,
NPEPPS,
UBE3A,
NUP43,
KCNH8,
CACNA1A,
CKB,
CHI3L1,
VCP,
RCOR1,
PRL,
VRK2,
PROS1,
NT5C2,
PSMC6,
CASP1,
H3P30,
TM7SF2,
PIK3CB,
ELK1,
MTDH,
EPHB2,
HDAC3,
PTPN5,
TGM1,
NRF1,
BDNF-AS,
FAP,
HYPK,
PRKCA,
MAPK11,
AHSG,
ADA,
MGP,
PRKAB1,
MCF2L,
KHDRBS1,
MAP3K5,
RGS2,
SHBG,
FOXP1,
MFAP1,
MOK,
CXCR6,
P4HB,
PVALB,
MAP2,
DCTN4,
ADCYAP1,
TPPP,
PHGDH,
LGALS3,
NPY2R,
ALPP,
TREM2,
RAB11A,
LRP2,
SLC1A1,
NGFR,
NGF,
PRNP,
ALOX5,
EIF2AK2,
NTF4,
PRKAA1,
MMP2,
MTHFR,
ACP3,
TUSC2,
NCAM1,
PYCARD,
RASD2,
CYP46A1,
S100A12,
FAM193A,
AGT,
MRPS30,
SGSM3,
TNFRSF11B,
NUP62,
REG3A,
REN,
SERPINE1,
ADRA1A,
PSAT1,
UBQLN1,
RXRA,
PRKAA2,
PDAP1,
BECN1,
FIS1,
IFNL4,
MRC1,
NF2,
PLAG1,
AGO2,
PIN1,
PAPOLA,
ASAP1,
KCNIP3,
ADRA2B,
CCHCR1,
ASAP2,
CALB2,
SLC2A1,
DECR1,
TNNI3,
SLCO6A1,
ATN1,
TIMP2,
LPAR2,
TIMP1,
EDNRA,
EGF,
EGR1,
GRIN3A,
EPHA3,
UBXN11,
TGFB1,
TFRC,
FCGR3A,
FCGR3B,
FEN1,
FGF9,
FGFR3,
FRAXE,
NR5A1,
KALRN,
SELENBP1,
MSTN,
MFN2,
BRS3,
TRPC5,
GLO1,
RLS1,
CASP2,
CASP8,
CBS,
VWF,
CD28,
MIR155,
KL,
ADIPOQ,
MIR132,
VDR,
MIR10B,
GPR52,
ENHO,
GSTK1,
MIR34A,
CP,
TSPO,
UBC,
TYMS,
TWIST1,
CCN2,
USP12,
CUX1,
CX3CR1,
TTR,
METRN,
HTR1A,
IAPP,
ACKR3,
KLK3,
STIM2,
IFNG,
KIDINS220,
HMOX1,
APRT,
HP,
TOMM40,
GTF2H1,
SOD2,
HSPD1,
MEG3,
ZBTB16,
ATF7IP,
APEX1,
APAF1,
MTPAP,
HACE1,
IFNA2,
GSK3B,
GPR42,
ACE2,
SPHK1,
ITPR3,
IFT57,
SSTR4,
NGB,
ATG7,
IER3,
SPON2,
HOMER1,
CPQ,
GDF15,
ATF5,
NLRP1,
LIN7A,
TUBB4B,
TRIP10,
SPTLC1,
RPH3A,
UBD,
GSTO1,
AHSA1,
NREP,
CD163,
CCT2,
CAP1,
SORBS1,
KLF4,
HAND1,
MLXIP,
GRAP2,
CIB1,
PRMT5,
APLN,
SEMA3A,
CERS1,
PSIP1,
PDIA6,
CDK5R1,
WDR5,
ARFGEF2,
BCL10,
SOCS3,
WWP1,
IMMT,
WASHC5,
NR1H3,
SV2B,
SV2A,
SBNO2,
BTRC,
CHEK2,
CCL27,
PNKP,
BCAR1,
APC2,
MGLL,
BAG3,
MACROH2A1,
CCL4L2,
HDAC6,
SGPL1,
LZTS3,
GABBR2,
CLOCK,
CPLX2,
HACD1,
DNAJA2,
PSME3,
KEAP1,
NAT2,
SV2C,
MARCHF8,
IL34,
HJV,
PDIK1L,
ERFE,
ZDHHC15,
OXER1,
PTCRA,
SCAMP5,
GPRC6A,
EHMT1,
STH,
IPMK,
GPX6,
MRGPRX1,
OR10A4,
GOLGA8G,
RAB7B,
MCIDAS,
CFAP54,
GPR151,
PPARGC1B,
CD200R1,
ASRGL1,
TET1,
UBXN6,
SLC25A28,
MAGT1,
EVA1A,
CYSTM1,
DNAJC14,
DCLK3,
FOXP2,
OPN4,
NLRP3,
CARD16,
MRGPRX3,
MRGPRX4,
GSTO2,
OSR1,
NPSR1,
CCL4L1,
CIMT,
POU5F1P3,
POU5F1P4,
MIR421,
MIR549A,
MIR615,
CASC8,
PSS,
ZGLP1,
MIR942,
TIMM23,
MIR1247,
ECT,
TUNAR,
TMX2-CTNND1,
MIR4488,
GDNF-AS1,
CBSL,
LINC02605,
MT1IP,
MIR486-1,
MIR106A,
MIR146B,
MIR122,
MIR128-1,
MIR146A,
MIR150,
MIR200A,
MIR200C,
MIR21,
MIR214,
MIR22,
MIR222,
MIR26B,
MIR27A,
MIR302A,
MIR34B,
VN1R17P,
GPR166P,
MIR196B,
PANK2,
RTL10,
TBC1D9,
GIT1,
NSG1,
NAAA,
IL37,
DISC1,
PDLIM3,
HPGDS,
HTRA2,
SLCO1B3,
FLVCR1,
MTHFSD,
SETD2,
DROSHA,
MYLIP,
UBQLN2,
PACSIN1,
SLC40A1,
CHCHD2,
AADAT,
SIGLEC7,
MLH3,
AKAP8L,
FGF21,
ACSBG1,
RRS1,
PHLPP1,
CRTC1,
DICER1,
ABCB10,
BRD4,
LPAR3,
ARFIP2,
IL17RA,
QPCT,
CHRDL2,
RNF19A,
SOSTDC1,
NGDN,
ATRNL1,
POLDIP2,
HSPA14,
UBR5,
NCKIPSD,
CMTR2,
GPRC5C,
ZNF395,
SPHK2,
UBQLN4,
SLC12A9,
S100A14,
SLC12A5,
SORCS2,
CFAP97,
GBA2,
HES4,
CTDSP1,
LGR6,
PAPPA2,
SLC39A8,
LIN7B,
ZMAT3,
HDAC8,
PRPF40A,
SIRT6,
GPRC5D,
NUB1,
TNFRSF6B,
HDL3,
S1PR5,
IMMTP1,
DDIT4,
UGT1A1,
TMEM106B,
TMED9,
AHI1,
TUG1,
ATG16L1,
MAP1S,
SBNO1,
ATAD3A,
NAT10,
FBXW7,
GHRL,
CXCL12,
RIPK2,
DRD5,
GATA4,
GC,
GH1,
GIP,
GJA1,
GLP1R,
GPR3,
GPX4,
GPX7,
GRN,
GRIK5,
GRIN1,
GRINA,
GRM2,
CXCL3,
GYS1,
GZMB,
HCLS1,
HDAC2,
GAD2,
GABRA2,
XRCC6,
F13B,
DUSP2,
E2F2,
EMP1,
ERCC6,
ERN1,
EZH1,
F11,
F13A1,
FABP4,
FSHMD1A,
PTK2B,
FBN1,
FGF2,
FGF13,
FGFR2,
FLNA,
FMR1,
MTOR,
HIF1A,
UBE2K,
HLA-C,
LBP,
IL18,
ITGA2B,
JAG2,
KCNA1,
KCNA5,
KCNA6,
KDR,
KPNA1,
LBR,
IL13,
LCN2,
LIMK1,
LMNA,
LNPEP,
LPA,
LPL,
LTB,
SMAD3,
IL17A,
IL9,
HLA-DPB1,
TNC,
HMGB1,
HNF4A,
HPCA,
HRES1,
DNAJB2,
DNAJA1,
HSPA8,
DNAJB1,
IDH1,
IL7,
IDUA,
IFNA1,
IFNA13,
IKBKB,
IL1A,
IL2RB,
IL4,
IL4R,
DTX1,
DRD3,
IRS2,
DPYSL2,
ATP2A2,
ALDH7A1,
ATRX,
B2M,
BAG1,
BCHE,
BCL2L1,
TNFRSF17,
BGLAP,
BNIP3,
C3,
C5,
C5AR1,
CAD,
CALD1,
CAMK2A,
CAST,
CAV1,
CD14,
ATHS,
ATF4,
STS,
ADCYAP1R1,
ABCA1,
ASIC1,
ACHE,
ACO1,
ACO2,
ACVR2B,
ACVRL1,
ADAM10,
ADM,
AQP9,
ADORA2B,
PARP1,
ABCD1,
ALPI,
ANGPT2,
ANK1,
ANXA3,
XIAP,
CD34,
CD40,
CD47,
DCT,
CSH2,
CTAA1,
CTNND1,
CYBB,
CYP3A4,
DAB2,
DAXX,
DBH,
DDIT3,
CSF1R,
DHCR24,
DHFR,
DLG1,
DLG3,
DMD,
DNAH6,
DNASE1,
DPP6,
CSH1,
CSE1L,
CDK9,
CNTN1,
CDKN1A,
CDKN1B,
CDKN2A,
CDX2,
CETP,
CHAT,
CHRM1,
CLCN1,
COL4A2,
MAPK14,
COL11A2,
COMT,
KLF6,
ATF2,
CRH,
CRK,
CRYAB,
CS,
MBL2,
MC1R,
MCL1,
MECP2,
SLCO2A1,
SLPI,
SUMO3,
SUMO2,
SNAP25,
SNCB,
SNCG,
SORL1,
SOX2,
SOX4,
SP1,
SPN,
SPP1,
SPTBN2,
SRI,
STAT5A,
STAT5B,
STATH,
SUPT4H1,
SLC12A2,
SLC12A1,
SLC9A5,
MAP2K4,
RPS6KA1,
RPS6KB1,
RPS19,
RRAS,
SORT1,
SCN4A,
CCL4,
SELENOP,
SGCA,
SLC9A1,
SGK1,
SH3GL3,
SKIL,
SKP1,
SLC1A3,
SLC1A6,
SLC2A4,
SLC6A3,
SYN1,
TAF4,
TARBP2,
ULK1,
AIMP2,
COIL,
MIA,
KDM5C,
KDM5D,
BAP1,
FZD4,
LOH19CR1,
CUL1,
RAB7A,
DGKE,
BLZF1,
PIAS1,
KHSRP,
PSMG1,
HSD17B6,
AOC3,
PDE5A,
ST8SIA4,
XPC,
TCF3,
TRPC1,
TFAM,
TGM3,
TH,
NKX2-1,
CLDN5,
TNFRSF1B,
TRAF6,
TRP-AGG2-6,
TRPC4,
XBP1,
TXNRD1,
SUMO1,
UCP1,
UCP2,
KDM6A,
VCL,
VIP,
VIPR2,
ROS1,
ROCK1,
RNASE4,
NFATC4,
MTRR,
MUTYH,
PPP1R12A,
NAIP,
NDUFA1,
NEFH,
NEK1,
NEUROD1,
NNAT,
MTNR1A,
NOS1,
NOS2,
PNP,
NPC1,
NPM1,
NPPA,
NPY5R,
SLC11A2,
MTR,
CYTB,
NT5E,
MSX1,
MAP3K1,
KITLG,
MLF1,
FOXO4,
MMP14,
MNAT1,
CD200,
MSN,
MT1A,
MT1X,
MT1B,
MT1E,
MT1F,
MT1G,
MT1H,
MT1JP,
MT1M,
MT1L,
NRGN,
NTF3,
RLN2,
PTMA,
MAPK8,
MAPK10,
MAP2K1,
MAP2K2,
MAP2K6,
PSEN1,
PSG5,
PTBP1,
PTMAP4,
PKIB,
PTPN13,
RAC1,
RAF1,
RAN,
RANBP2,
RARB,
RENBP,
RET,
PKN2,
PPT1,
OPA1,
PCP4,
P2RX7,
PAEP,
PAM,
PRKN,
PC,
PCBP1,
PCM1,
PCNA,
PDE1B,
POU5F1,
PDK3,
PEX7,
PF4,
PGD,
SERPINB6,
PITX3,
PLCG1,
PMS2,
H3P10
Drugs
2'-O-(2-methoxyethyl) modified oligonucleotide targeted to huntingtin RNA,
2'-O-methyl phosphorothioate RNA oligonucleotide, 5'-m5CUGm5CUGm5CUGm5CUGm5CUGm5CUGm5CUG-3',
2,3,4,5 tetrahydro-2,8-dimethyl-5-[2-(6-methyl-3-pyridinyl)ethyl]-1H-pyrido[4,3-b]indole dihydrochloride
2'-O-(2-methoxyethyl) modified oligonucleotide targeted to huntingtin RNA,
2'-O-methyl phosphorothioate RNA oligonucleotide, 5'-m5CUGm5CUGm5CUGm5CUGm5CUGm5CUGm5CUG-3',
2,3,4,5 tetrahydro-2,8-dimethyl-5-[2-(6-methyl-3-pyridinyl)ethyl]-1H-pyrido[4,3-b]indole dihydrochloride,
2-(3,7-Dimethyl-octa-2, 6-dienyl)-6-ethylamino-3-hydroxy-5-pentyl-[1,4]benzoquinone,
5,7-dichloro-2-dimethylaminomethyl-8-hydroxyquinoline hydrochloride,
5,7-dichloro-2-methylaminomethyl-8-hydroxyquinolone hydrochloride,
5-bromo-N-(prop-2-yn-1-yl)-2-(1H-1,2,4-triazol-1-yl) pyrimidine-4,6-diamine,
AASSGVSTPGSAGHDIITEQPRS (P42 peptide),
Adeno-associated viral vector serotype 5 encoding a microRNA targeted to human huntingtin gene,
Adeno-associated viral vector serotype rh10 containing the human cholesterol 24-hydroxylase gene,
Carbenoxolone,
Cysteamine bitartrate
(
CYSTAGON
),
Deutetrabenazine,
Ethyl eicosopentaenoate,
Lithium citrate tetrahydrate (in reverse-micelle formulation),
Phosphodiesterase 10 (PDE10) Inhibitor,
Porcine fetal neural gabaergic cells and/or precursors aseptically prepared and coated with anti-Mhc-1 Ab for intracerebral implantation,
Porcine fetal neural gabaergic cells and/or precursors aseptically prepared for intracerebral implantation for Huntington's disease,
Pridopidine,
Selisistat,
Tyr-Gly-Arg-Lys-Lys-Arg-Arg-Gln-Arg-Arg-Gly-Gly-Asp-Leu-Leu-Pro-Arg-Gly-Ser,
phenol, 4-[2-(aminomethyl)-4-thiazolyl]-2,6-bis(1,1-dimethylethyl) monohydrochloride
Registered!
Huntington disease (HD) is an inherited condition that causes progressive degeneration of neurons in the brain. Signs and symptoms usually develop between ages 35 to 44 years and may include uncontrolled movements, loss of intellectual abilities, and various emotional and psychiatric problems. People with HD usually live for about 15 to 20 years after the condition begins. It is caused by changes (mutations) in the HTT gene and is inherited in an autosomal dominant manner. Treatment is based on the symptoms present in each person and may include various medications.
There is also a less common, early-onset form of HD which begins in childhood or adolescence. For more information on this form, please visit GARD's juvenile Huntington disease Web page.
There is also a less common, early-onset form of HD which begins in childhood or adolescence. For more information on this form, please visit GARD's juvenile Huntington disease Web page.