Familial Monosomy 7 Syndrome
Watchlist
Retrieved
2021-01-23
Source
Trials
—
Genes
—
Drugs
2-hydroxymethyl-2-methoxymethyl-1-azabicyclo[2,2,2]octan-3-one,
Allogeneic ex vivo expanded umbilical cord blood cells,
Allogeneic multi-virus specific T lymphocytes targeting BK virus, cytomegalovirus, human herpesvirus-6, Epstein Barr virus and adenovirus
2-hydroxymethyl-2-methoxymethyl-1-azabicyclo[2,2,2]octan-3-one,
Allogeneic ex vivo expanded umbilical cord blood cells,
Allogeneic multi-virus specific T lymphocytes targeting BK virus, cytomegalovirus, human herpesvirus-6, Epstein Barr virus and adenovirus,
Arsenic trioxide
(
Arsenic Trioxide Accord,
Arsenic trioxide Mylan,
Arsenic trioxide medac,
TRISENOX
),
Asunercept,
Azacitidine
(
AZACITIDINE ACCORD,
AZACITIDINE BETAPHARM,
AZACITIDINE CELGENE,
AZACITIDINE MYLAN,
VIDAZA
),
Decitabine
(
DACOGEN
),
Fully human IgG1 antibody specific for CD33,
Humanised IgG4 monoclonal antibody to the human toll-like receptor type 2,
Imatinib mesilate
(
GLEEVEC,
GLIVEC,
IMATINIB TEVA,
IMATINIB TEVA B.V.
),
Lenalidomide
(
LENALIDOMIDE ACCORD,
REVLIMID
),
Lintuzumab,
Luspatercept
(
REBLOZYL
),
Magrolimab,
Mocetinostat,
NEDD8-activating enzyme (NAE) inhibitor,
Rigosertib,
Sapacitabine,
Sotatercept,
Stem/progenitor cells derived from ex vivo expanded allogeneic umbilical cord blood cells,
Thalidomide
(
THALIDOMIDE CELGENE,
THALOMID
),
Val-Leu-Gln-Glu-Leu-Asn-Val-Thr-Val (Pr1 nanopeptide, sequence 169-177, of proteinase 3),
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 neoplastic disease characterized by infantile to childhood onset of evidence of bone marrow insufficiency/failure associated with increased risk for myelodysplastic syndrome or acute myeloid leukemia. Most patients present with petechiae, easy bruising, or anemia. Rapid progression is common, and prognosis is generally poor.