RDR Alert®

Lonafarnib was linked to improved mortality over a maximum of 11 years follow-up, according to supporting data.

A discrepancy regarding a pre-licensing inspection of a third-party manufacturing facility led to the Complete Response Letter.

The study presented at AAO provides an update on previously shared 6-month data from earlier this year.

The estimated the prevalence of C diff infections is about 40 in 30,000 for the sickle cell disease group and about 5000 in 11.8 million for the non-sickle cell disease group.

Identifying early stages of chronic kidney disease in patients with sickle cell anemia has the potential of allowing for personalized treatment and better prognosis.

A new study finds patients with sickle cell disease are moderately adherent to hydroxyurea.

The label now indicates hydroxyurea use does not affect benefit received from the medication.

A review of studies seeks the prevalence, determinants and outcomes of iron deficiency anemia in expecting mothers with sickle cell disease.

New phase 1 data show the first-in-class oral therapy is not affected by the burden on metabolism via hepatic or renal disease.

In data presented during Kidney Week, researchers find the risk of developed an acute kidney injury increased in both sickle cell trail and sickle cell disease patients.

According to recommendations, electronic media should actively engage in efforts to address the prevention and management of sickle cell disease.

The Full Field Peripheral Blood Smear application addresses shortcomings of current digital solutions.

A common theme among clinical staff focus groups was the pervasiveness of systemic and social barriers to addressing basic needs in children with sickle cell disease.

Stem cell transplantation can be a curative treatment for sickle cell disease, but there is some safety risks.

Multi-center, sickle cell studies may face a host of barriers and challenges in achieving target sample sizes.

Venetoclax in combination with azacitidine was shown to prolong survival and increase chance of remission.

New findings from the Congo highlight risks both mothers and their children face in delivery.

The orphan drug will be the first ever FDA-approved granular hydrocortisone formulation specifically developed for pediatric patients.

The complexities associated with the disease and lack of validated risk predictors makes such a model challenging to implement.

Mepolizumab will be used to treat both adult and pediatric patients with HES for at least 6 months without an identifiable non-hematologic secondary cause.

A study found that patients more commonly self-reported unintentional barriers to treatment adherence—such as forgetfulness and external influences—than intentional ones.

The treatment could also be used for Alzheimer disease and mid-stage Huntington disease for patients who suffer from greater cognitive deficits.

These discrepancies are more pronounced in young, adolescent females and older adolescent males.

Gene addition and editing strategies have potential of curing sickle cell disease and transfusion-dependent thalassemia, but there are still many obstacles that must be overcome.

Interview results show patients can generally recall giving transfusion consent, but not information on the procedures benefits and risks.