Stat3-Related Early-Onset Multisystem Autoimmune Disease
Watchlist
Retrieved
2021-01-23
Source
Trials
—
Genes
STAT3,
IL23R,
LRRK2,
RMI2,
C1QTNF6,
ANKRD30A,
KCNH7,
NKD1,
NEURL4,
ARID5B,
ANKRD55,
CARD9,
ERAP2,
BACH2,
PLEKHA1,
ARHGAP31,
ZMIZ1,
ATG16L1,
UBASH3A,
CPT1C,
ADCY7,
PTPN22,
PUS10,
LINC02341,
LINC00993,
PTCSC2,
LINC00824,
LINC01250,
MIR3681HG,
LINC00271
STAT3,
IL23R,
LRRK2,
RMI2,
C1QTNF6,
ANKRD30A,
KCNH7,
NKD1,
NEURL4,
ARID5B,
ANKRD55,
CARD9,
ERAP2,
BACH2,
PLEKHA1,
ARHGAP31,
ZMIZ1,
ATG16L1,
UBASH3A,
CPT1C,
ADCY7,
PTPN22,
PUS10,
LINC02341,
LINC00993,
PTCSC2,
LINC00824,
LINC01250,
MIR3681HG,
LINC00271,
INS-IGF2,
IRF1-AS1,
C1orf141,
LINC02649,
C12orf42,
LURAP1L,
CCDC88B,
CUTALP,
SPATA13,
MB21D2,
IGF2-AS,
TMEM131,
ALDH2,
CRB1,
RAB5C,
RAB5B,
PTPN2,
PSMD5,
TNFRSF11B,
NTRK1,
SMAD3,
LPP,
JAK1,
IRF5,
IL2RA,
GPR35,
FUT2,
FLT3,
FAP,
DAG1,
CAMK4,
SLC22A5,
STAT4,
SUOX,
RASGRP1,
ADGRL2,
FNBP1,
ATXN2L,
CD226,
VAV3,
DLEU1,
PLXNC1,
SH2B3,
TG,
TNFSF15,
ELMO1,
SH2D2A,
CCN4,
UBE2L3,
TYK2,
TPO,
LINC02357
Drugs
A highly purified formulation of Staphylococcus aureus protein A,
Allogeneic multi-virus specific T lymphocytes targeting BK virus, cytomegalovirus, human herpesvirus-6, Epstein Barr virus and adenovirus,
PEGylated peptide inhibitor of complement C3
A highly purified formulation of Staphylococcus aureus protein A,
Allogeneic multi-virus specific T lymphocytes targeting BK virus, cytomegalovirus, human herpesvirus-6, Epstein Barr virus and adenovirus,
PEGylated peptide inhibitor of complement C3,
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!
A rare, genetic, lypmhoproliferative syndrome characterized by early onset recurrent infections, lymphadenopathy with hepatosplenomegaly and variabe autoimmune disorders, including hemolytic anemia, thrombocytopenia, neutropenia, enteropathy, type I diabetes, scleroderma, arthritis, atopic dermatitis, and inflammatory lung disease. Patients commonly have failure to thrive. Variable immunologic findings include decreased regulatory T-cells, hypogammaglobulinemia, and reduction in memory B cells.