Preferred Label : Cimdelirsen;
NCIt synonyms : GHR Antisense Oligonucleotide IONIS-GHR-LRx; LICA Drug IONIS-GHR-LRx; Ligand-conjugated Antisense Drug IONIS-GHR-LRx; Growth Hormone Receptor Antisense Oligonucleotide IONIS-GHR-LRx;
NCIt definition : An antisense oligonucleotide targeting the growth hormone receptor (GHR; somatotropin
receptor) mRNA, with potential somatotropin-inhibiting activity. Upon subcutaneous
administration, cimdelirsen hybridizes with GHR mRNA, which blocks translation of
the GHR protein. Reduction of GHR levels in turn decreases the circulating level of
insulin-like growth factor-1 (IGF-1). IGF-1 is a hormone that plays an important role
in childhood growth and has anabolic effects in adults. Excess production of IGF-1
results in various disease states including acromegaly.;
UNII : F593G1C1YO;
CAS number : 2131025-82-4;
Molecule name : IONIS-GHR-LRx; IONIS GHR LRX; ISIS-766720;
NCI Metathesaurus CUI : CL973173;
Origin ID : C162807;
UMLS CUI : C5554567;
Semantic type(s)
- concept_is_in_subset
- has_target
