Fragile X Syndrome
Watchlist
Retrieved
2022-04-26
Source
Trials
—
Genes
FMR1,
APP,
AFF2,
FMR1-IT1,
NUFIP2,
GRM5,
FRAXA,
FRAXE,
MMP9,
ACTB,
ARSD,
PVALB,
BDNF,
FXN,
FXR1,
FMR1-AS1,
G6PD,
FXR2,
PGD,
RBMS3,
CYFIP2,
SRRM2,
PNO1,
MFAP1,
MAK16,
EIF4E,
F9,
PIK3CA,
APRT,
PIK3CD
FMR1,
APP,
AFF2,
FMR1-IT1,
NUFIP2,
GRM5,
FRAXA,
FRAXE,
MMP9,
ACTB,
ARSD,
PVALB,
BDNF,
FXN,
FXR1,
FMR1-AS1,
G6PD,
FXR2,
PGD,
RBMS3,
CYFIP2,
SRRM2,
PNO1,
MFAP1,
MAK16,
EIF4E,
F9,
PIK3CA,
APRT,
PIK3CD,
PIK3CG,
PTBP1,
LINC01672,
RAC1,
PIK3CB,
MECP2,
IGF2,
IL6,
ST14,
NCS1,
SDC2,
MLH1,
CYFIP1,
RPS6KB1,
MSH2,
NR1H4,
VEGFA,
PDE4D,
SHANK1,
FOSL1,
IDS,
LIMK1,
SOD1,
BMPR2,
GRIK1,
RIC8A,
CD44,
GRM1,
CPEB1,
PCSK9,
DLG3,
RABEP2,
KIAA1109,
HSPG2,
C9orf72,
RSS,
SLC36A1,
ST8SIA4,
DGKK,
WNT7A,
PTPN5,
VIP,
USF2,
MIR219A1,
PKP4,
USF1,
UBE3A,
TWIST1,
MIR510,
ICAM5,
SYN1,
STXBP1,
STATH,
C20orf181,
SRY,
SPARC,
MAGT1,
YTHDF2,
WASF1,
DICER1,
CHMP4A,
INPP5K,
MED18,
CHD7,
MBD5,
NBEA,
SHC2,
TWNK,
SNRPN,
BRD4,
TARDBP,
SIRT1,
ARHGEF9,
TOP3B,
ADARB1,
KCNT1,
CLSTN1,
DSTN,
RAI1,
CTCF,
PDLIM5,
ABCB6,
HDAC6,
MED12,
PCA3,
NRXN1,
TDRD3,
APOA1,
SMS,
HTC2,
GRN,
GRIN2A,
ADCYAP1,
GSK3B,
GSN,
HTT,
NRG1,
HTR2A,
SLC12A2,
IAPP,
IGF1,
IGFALS,
IRF6,
KCNH1,
KCNQ2,
LGALS4,
GRIA2,
GABRD,
GABPA,
AKT1,
AVP,
BCR,
BRCA2,
CAT,
CD47,
CRHR1,
DLD,
DLG4,
DSCAM,
RCAN1,
ELAVL2,
FBN1,
ANG,
ALPP,
ALPI,
LMNA,
CAPRIN1,
MAOA,
PDE2A,
ADAM10,
PPP2R5E,
MAPK3,
PTEN,
ANXA1,
RANGAP1,
REST,
RGS4,
ATXN8OS,
SCN2A,
SHBG,
SKI,
SLC1A2,
SLC6A4,
SLC6A8,
CFP,
PAX3,
MAS1,
PAK1,
MBS1,
MDM2,
MEF2A,
ATXN3,
ADCY1,
PPP1R12A,
NF1,
NFE2L1,
NFE2L2,
NHS,
NNAT,
NOS1,
NRF1,
NTRK2,
NUP98,
PLCG1
Drugs
(3S)-(+)-(5-chloro-2-methoxyphenyl)-1,3-dihydro-3-fluoro-6-(trifluoromethyl)-2H-indol-2-one,
Acamprosate calcium,
Alpha-tocopherol and ascorbic acid
(3S)-(+)-(5-chloro-2-methoxyphenyl)-1,3-dihydro-3-fluoro-6-(trifluoromethyl)-2H-indol-2-one,
Acamprosate calcium,
Alpha-tocopherol and ascorbic acid,
Balipodect,
Cannabidivarin,
Mavoglurant,
Metadoxine,
N-[(1R)-1-phenylethyl]-6-{1H-pyrazolo[3,4-d]pyrimidin-4-yl}quinazolin-2-amine,
R-baclofen,
Trofinetide,
tideglusib
Registered!
Fragile X syndrome is a genetic condition involving changes in part of the X chromosome. This condition causes a range of developmental problems including learning disabilities and cognitive impairment. It is the most common form of inherited intellectual disability in males and a significant cause of intellectual disability in females. Other signs and symptoms may include symptoms of autism spectrum disorders, seizures, and characteristic physical features. Fragile X syndrome is caused by a change (mutation) in the FMR1 gene and is inherited in an X-linked dominant manner. There is no cure yet and treatment is based on the symptoms present in the person. Early physical and education therapy is recommended.