Disease-modifying treatments (DMTs) can help lessen pain from low blood oxygen levels. And two new, FDA-approved gene therapies may help end hospitalizations for SCD pain forever.
Pediatric Research publishes original papers, invited reviews, and commentaries on the etiologies of diseases of children and disorders of development, extending from molecular biology to epidemiology. Use of model organisms and in vitro techniques relevant to developmental biology and medicine are ...
In 2023, 2 different gene therapies were approved for individuals with severe sickle cell disease (SCD). The small number of patients treated on the pivotal clinical trials that led to these approvals have experienced dramatic short-term reductions in the occurrence of painful vaso-occlusive crises...
Fifty years ago, people with sickle cell disease (SCD) were discouraged from becoming pregnant, but now, most should be supported if they choose to pursue a pregnancy. They and their providers, however, should be aware of the physiological changes of pregnancy that aggravate SCD and pregnancy’...
23-25 January 2013 Sickle cell disease is the most prevalent monogenic disor- der worldwide resulting from single DNA mutation in beta globin gene. The mapping on the pattern of its distri- bution in India has been studied to a great extent. How- ever paucity of adequate data throughout ...
Individuals with sickle-cell disease now have a greater chance of living to adulthood than ever before. That means you have a greater chance of car...
Since not all patients with sickle cell disease experience vaso-occlusion and the frequency of attacks can vary widely between patients, the MIT researchers hope that their findings may help them to devise a way to predict these crises for individual patients....
Improve Quality of Life: By addressing the root causes of degenerative diseases and injuries, stem cell therapy aims to enhance the overall quality of life for patients, providing relief from symptoms and potentially slowing disease progression. ...
Like other species, we are the products of millions of years of adaptation. Now we're taking matters into our own hands.
Retrospectively analyzing data from the Cooperative Study of Sickle Cell Disease (CSSCD) to calculate incidence rates for first-time VTE, Naik and colleagues found an incidence rate of 5.2 events per 1000 person-years (95% confidence interval [CI], 3.8-6.9) in 1523 SCD patients aged ≥15 ...