BCH-BB694 — an experimental gene therapy targeting the BCL11A gene — safely increased the levels of fetal hemoglobin and…
Articles by Marta Figueiredo, PhD
Six months of treatment with oral IMR-687 safely and effectively reduced the number of vaso-occlusive crises (VOCs) and other disease-associated…
Sickle Cell Disease News brought you daily coverage of key findings, treatment developments, clinical trials,…
CTX001, an experimental gene-editing cell therapy, safely and effectively increased the levels of fetal hemoglobin and prevented vaso-occlusive…
Starting treatment with rivipansel (GMI-1070) shortly after vaso-occlusive crisis (VOC) onset significantly shortened hospital stays and the time…
The first wave of COVID-19Â in Europe severely disrupted access to care and raised stress and anxiety in people with…
FT-4202, a potential disease-modifying oral treatment for sickle cell disease (SCD) by Forma Therapeutics, has been designated an …
The National Institutes of Health (NIH) has awarded a $7.1 million grant to University of Illinois Chicago (UIC) researchers to evaluate the…
The U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation to GlycoMimetics’ investigational therapy…
Bluebird Bio’s investigational gene therapy LentiGlobin safely prevents serious vaso-occlusive crises and acute chest syndrome in adolescents…