RDR Alert®

Compared with controls, children and adolescents with sickle cell anemia were more likely to be underweight and exhibit signs of stunting and wasting.

Patients who received voxelotor 1500 mg achieved significant improvements in markers of hemolysis through week 72 when compared with the placebo group.

A new study supports the potential association between hydroxyurea and improved neurocognitive performance in pediatric patients.

The PCSK9 inhibitor was linked to 27% mean reduction in LDL-C over 12 weeks.

New phase 2 data from a 24-week assessment suggest the first-in-class drug could benefit exercise and pulmonary-vascular outcomes.

The new indication for the liposomal-combination therapy is based on safety outcomes from a pair of single-arm trials.

A budget impact analysis suggest the burgeoning drug class may not be as available as it could be for sickle cell patients.

Multiple myeloma is currently incurable with many patients experiencing relapses.

A proposed smartphone-based intervention to address hydroxyurea nonadherence was welcomed by a majority of surveyed individuals with sickle cell disease.

Pegcetacoplan, an investigational complement C3 inhibitor, demonstrated superiority to C5 inhibitor, eculizumab, for paroxysmal nocturnal hemoglobinuria.

Older age and higher education level were significantly linked with increased depression scores.

As a multi-systemic disorder, Fabry disease can impact the functions of various organs. As a genetic disorder, Fabry can impact the offsprings, parents, and relatives of the patient. Therefore, its effects are far-reaching.

A literature review identifies 3 broad factors that may contribute to bias in pain management of African Americans with SCD.

Few studies have specifically assessed the presentations and outcomes of sickle cell-related-leg ulcers in a female population.

A head-to-head assessment of the intravenous treatment options found greater efficacy for acetaminophen, supporting it more against the adverse effect-associated diclofenac.

Healthcare accessibility continues to be a significant barrier for patients and their families.

Number of red blood cell transfusions may be linked to decreased risk intrauterine fetal death.

Eric Wallace, MD, discusses Anderson-Fabry disease and the difficulties associated with screening, testing, and treatment.

The company suspended clinical trials of the gene therapy last month following patient diagnoses of acute myeloid leukemia and acute myelodysplastic syndrome.

Among 85 children with beta-thalassemia major and ≥5 years since primary vaccination, 23 were found to be seronegative.

Brain function improved with hydroxyurea treatment of sickle cell disease in prospective study with cerebrovascular and neuropsychologic measures.

A look back on key moments from Episode 1 of the Rare Disease Report podcast —featuring Kim Smith-Whitley, MD.

The FDA will consider the approval of the biologics license application next month.

Although overall decrease in mortality was similar to the control group, sickle cell populations were still less likely to receive a kidney transplant.

Perspective on how the interleukin-targeting biologic has unique benefit for the rare, under-resourced disease.