A phase 2 study of HQK-1001, an oral fetal haemoglobin inducer, in β-thalassaemia intermedia.
Lebanese American University, Beirut, Lebanon; Rafik Hariri University Hospital, Beirut, Lebanon.
Adlette
A
Inati
Mario
M
Kahale
Susan P
SP
Perrine
David H K
DH
Chui
Ali T
AT
Taher
Suzanne
S
Koussa
Therese
T
Abi Nasr
Hussein A
HA
Abbas
Richard G
RG
Ghalie
D000293:Adolescent; D000328:Adult; D002087:Butyrates / Q000008:administration & dosage*; D005260:Female; D005319:Fetal Hemoglobin / Q000096:biosynthesis* / Q000235:genetics; D006801:Humans; D008297:Male; D008875:Middle Aged; D055815:Young Adult; D017086:beta-Thalassemia / Q000097:blood / Q000188:drug therapy* / Q000235:genetics
D002087:Butyrates; D005319:Fetal Hemoglobin; C074677:2,2-dimethylbutyric acid
anaemia; fetal haemoglobin; gene expression; haemoglobinopathies; β-thalassaemia