Sickle Cell Anemia
Watchlist
Retrieved
2022-04-26
Source
Trials
—
Genes
HBB,
NFE2L2,
HP,
TNF,
VCAM1,
DHODH,
NPPB,
CAD,
CACNA1C,
UMPS,
HMOX1,
SPTA1,
BCL11A,
HBG2,
UGT1A8,
HBE1,
UGT1A10,
UGT1A4,
UGT1A7,
UGT1A6,
UGT1A3,
UGT1A9,
UGT1A5,
G6PD,
UGT1A1,
ARAP1,
OR51B5,
UBIAD1,
EDN1,
MYB
HBB,
NFE2L2,
HP,
TNF,
VCAM1,
DHODH,
NPPB,
CAD,
CACNA1C,
UMPS,
HMOX1,
SPTA1,
BCL11A,
HBG2,
UGT1A8,
HBE1,
UGT1A10,
UGT1A4,
UGT1A7,
UGT1A6,
UGT1A3,
UGT1A9,
UGT1A5,
G6PD,
UGT1A1,
ARAP1,
OR51B5,
UBIAD1,
EDN1,
MYB,
OR51I2,
MCC,
RN7SL263P,
SELP,
LINC01847,
PGF,
MUC4,
MTHFR,
NPRL3,
RABEP1,
HBG1,
CD34,
HBA2,
HBA1,
GABPA,
SORCS2,
NOS3,
HBS1L,
F5,
HPX,
SCD,
RHCE,
ALB,
SELL,
VWF,
APOL1,
MBL2,
RHD,
F2,
CAT,
HAMP,
CSF3,
BCAM,
VEGFA,
REN,
F3,
EPO,
HPGDS,
CCR5,
UGT1A,
IL6,
HIF1A,
IL1B,
CXCL8,
HLA-DRB1,
SCT,
SELE,
SERPINE1,
ABO,
CYP2D6,
PSMA7,
GSTT1,
GSTK1,
FUT1,
KLF1,
DLL3,
IFNG,
IGF1,
CCL2,
SOD2,
TBX6,
IL4,
SLCO6A1,
TRV-AAC1-4,
CSF2,
TGFB1,
MIPEP,
ANXA5,
SPHK1,
HLA-DQB1,
ANXA1,
KL,
THBD,
AKT1,
GSTM1,
PLF,
HBD,
MIR144,
MPO,
APOB,
RAPGEF5,
AGT,
THBS1,
ANGPT2,
MYH9,
ICAM4,
LCN2,
ADRA1D,
CXCL10,
NT5E,
IL18,
PDE5A,
IL10,
MBTPS1,
ADCY6,
AGER,
ADRB2,
IL1A,
PPBP,
IGFBP3,
PRKAR1A,
TNFRSF1A,
GSTP1,
GCH1,
VPS51,
SCD5,
SAR1A,
ADAMTS13,
COMT,
FCGR3B,
RUNX2,
KDM1A,
ATP2A2,
ANTXR1,
CD14,
CAPN1,
HES7,
DECR1,
CD59,
CYP2C19,
RBM45,
CTLA4,
CD38,
TFPI,
CPLANE1,
PRDX2,
LIN28A,
NOD2,
CDH23,
PRRT2,
UGGT1,
CHD7,
CAMKMT,
KLF10,
NLRP3,
GALNT13,
TIE1,
TK2,
APOA5,
TLR2,
CCR2,
MIR454,
TNFSF15,
TALDO1,
TACR1,
KRT88P,
ROS1,
RPS19,
RYR1,
KRT90P,
LOC107987479,
SCN2A,
HOPX,
CCL5,
CCL7,
CX3CL1,
MPIG6B,
CBSL,
SFTPD,
SLC12A4,
SLC14A1,
SLCO1A2,
SMPD1,
SPG7,
SPRR2A,
CD24,
LPCAT1,
SLC35A2,
TRIM21,
TAC1,
SPTBN1,
NCF1,
RIPPLY2,
UGT2B7,
PER2,
MIR326,
CBX3,
ZPR1,
MBD2,
TRPA1,
RSPH1,
SPIN1,
HPSE,
SKA2,
ZFYVE9,
LIPG,
GRAP2,
DMRT2,
MIR301A,
MIR214,
AHSA1,
KAT5,
VHLL,
LINC01194,
GDF15,
APOBEC3B,
TRIM13,
LPCAT3,
CEBPZ,
NR1I3,
PIEZO1,
APLN,
SMUG1,
DIANPH,
BEAN1,
KEAP1,
CPVL,
MESP2,
TRPV1,
EOS,
HBFQTL2,
AIMP2,
CXADRP1,
AKAP1,
BRAP,
LOH19CR1,
PLA2G6,
PRRX2,
NANOS1,
AHSP,
OR10A4,
ICOS,
IL37,
HAVCR1,
TNFSF14,
SLC17A5,
TNFRSF6B,
POLDIP2,
RNF19A,
CLDN15,
PROZ,
LRSAM1,
ABCA1,
MOK,
CLDN3,
CYBB,
CXADR,
CTSK,
CST3,
MAPK14,
CRYZ,
CRP,
CRK,
COX8A,
CD40LG,
COL4A1,
CCR4,
CLCN7,
CHM,
CHIT1,
CTSC,
CDA,
CD63,
CD55,
ACE,
CYB5R3,
DNASE1,
FOXO3,
FKBP4,
FH,
FES,
FDPS,
FCGR3A,
FCGR2A,
FAAH,
ETFA,
EPAS1,
ELAVL2,
ELANE,
EGR1,
S1PR1,
ATN1,
DRD3,
DRD2,
CD47,
CD36,
FOSB,
AMBP,
ARG1,
ABCC6,
FAS,
APOE,
APOA1,
BIRC3,
BIRC2,
AMH,
ALOX5,
CD28,
ALAS2,
AKT2,
ADORA2B,
ADCY9,
ADA,
ACP3,
ACP5,
ACHE,
ARR3,
ASS1,
ATP2B4,
AVPR1A,
CD19,
CD6,
CD1C,
CD1B,
CD1A,
KRIT1,
CBS,
CASR,
CASP3,
CASP1,
CALM3,
CALM2,
CALM1,
BTK,
BRCA1,
BDNF,
BAX,
FOS,
MTOR,
PTX3,
MYH2,
P2RY6,
OXCT1,
OLR1,
OAT,
NOS1,
NM,
NHS,
NGF,
MUC1,
LFNG,
MST1R,
MFGE8,
MCL1,
MAP6,
SMAD2,
SMAD1,
LYZ,
LTB,
P4HB,
PAPPA,
PER1,
PF4,
PTH,
PRNP,
MAPK8,
MAPK1,
PRKCB,
PRKCA,
PPARG,
PPARA,
PON3,
PON1,
PLXNA2,
PLG,
PLA2G4A,
PLA2G2A,
PLA2G1B,
SERPINA1,
ABCB1,
LGALS3,
LDLR,
FUCA2,
GYPA,
HLA-G,
HLA-E,
HLA-A,
HFE,
CFH,
HEXA,
SERPIND1,
GZMM,
GTF2I,
LCAT,
GSR,
CXCL1,
NR3C1,
CBLIF,
GH1,
GAPDH,
GAD1,
ACKR1,
HMGB1,
HMGCR,
HSP90AA1,
ICAM1,
LAMC2,
KRT12,
KLRC3,
KCNN4,
JUND,
JUNB,
JUN,
ITGB2,
ITGAL,
ITGA4,
INSIG1,
IMPA1,
IL17A,
CXCR2,
IL5,
IL2,
IGF1R,
HBB-LCR
Drugs
(S)-3'-(OH)-desazadesferrithiocin-polyether, magnesium salt,
2,2-dimethylbutyric acid, sodium salt,
4,5-dihydro-2-(2,4-dihydroxyphenyl)-4-methylthiazole-4(S)-carboxylic acid
(S)-3'-(OH)-desazadesferrithiocin-polyether, magnesium salt,
2,2-dimethylbutyric acid, sodium salt,
4,5-dihydro-2-(2,4-dihydroxyphenyl)-4-methylthiazole-4(S)-carboxylic acid,
5-hydroxymethyl-2-furfural,
Allogeneic multi-virus specific T lymphocytes targeting BK virus, cytomegalovirus, human herpesvirus-6, Epstein Barr virus and adenovirus,
Autologous CD34+ haematopoietic stem cells transduced with lentiviral vector encoding the human betaA-T87Q-globin gene
(
ZYNTEGLO
),
Autologous CD34+ hematopoietic stem cells transduced with LentiGlobin BB305 lentiviral vector encoding the human BA-T87Q-globin gene,
Benserazide hydrochloride,
Decitabine and tetrahydrouridine,
Deferasirox
(
EXJADE,
DEFERASIROX MYLAN
),
Deferiprone
(
FERRIPROX,
DEFERIPRONE LIPOMED
),
Docosahexaenoic acid ethyl ester,
Extract of Sorghum bicolour leaf, Pterocarpus osun stem, Piper guineense seed and Caryophylli flower,
Human erythrocytes encapsulating inositol hexaphosphate,
Human haptoglobin,
Humanised monoclonal antibody against P-selectin
(
ADAKVEO
),
Hydroxycarbamide
(
DROXIA,
HYDREA,
SIKLOS,
XROMI
),
L-Glutamine
(
ENDARI,
NUTRESTORE
),
Levoglutamide,
PDE9 Inhibitor,
Pegylated carboxyhaemoglobin,
Poloxamer 188,
Rivipansel,
Sevuparin,
Sirolimus
(
RAPAMUNE
),
Synthetic hepcidin,
Voxelotor
(
OXBRYTA
),
autologous CD34+ hematopoietic stem cells with a CRISPR-edited erythroid enhancer region of the BCL11A gene,
haematopoietic stem cells and blood progenitors umbilical cord-derived expanded with (1R, 4R)-N1-(2-benzyl-7-(2-methyl-2H-tetrazol-5-yl)-9H-pyrimido[4,5-b]indol-4-yl)cyclohexane-1,4-diamine dihydrobromide dihydrate
Registered!
Sickle cell anemia is a disease in which the body produces abnormally shaped red blood cells that have a crescent or sickle shape. These cells do not last as long as normal, round, red blood cells, which leads to anemia (low number of red blood cells). The sickle cells also get stuck in blood vessels, blocking blood flow. Signs and symptoms of sickle cell disease usually begin in early childhood and may include anemia, repeated infections, and periodic episodes of pain (called crises). This condition is caused by mutations in the HBB gene and is inherited in an autosomal recessive pattern. Treatment typically focuses on controlling symptoms and may include pain medicines during crises; hydroxyurea to reduce the number of pain episodes; antibiotics and vaccines to prevent bacterial infections; and blood transfusions. On July 7, 2017, the FDA in the United States approved the use of Endari (prescription grade L-glutamine) to reduce the number of sickle cell crisis. Endari is the first FDA approved treatment that is also available for children with sickle cell disease five years of age and older.