Sickle Cell

Niemann-Pick is highly heterogenous and is characterized by hypervariable manifestations across patients.

Excessive hemolysis and genetic factors may be associated with cholelithiasis.

A sickle cell expert discusses the shifting therapeutic landscape as well as a need to rethink hemoglobin level threshold guidelines for patients.

Patients who reported neurocognitive challenges were more likely to have reduced skills related to instrumental activities of daily living.

Sharon Hrynkow, PhD, joins the Rare Disease Report to discuss the ultra rare and heterogenous lysosomal storage disorder.

Improving health literacy among these individuals may have a significant and positive impact on the transition from pediatric to adult care.

A co-located model of care may be highly beneficial in easing the transition from pediatric to adult care.

A study notes that irradiation did not impact transfusion requirements or relevant laboratory values among patients receiving chronic blood transfusions.

In a single-center study, no patients were screened annually for HIV and HCV.

While the nerve disease is not life threatening, patients may experience considerable poor quality of life and emotional distress.

A new report sheds light on the gaps in knowledge and research surrounding the reproductive health of girls and women with sickle cell disease.

Adults who have received at least one prior line of therapy may receive daratumumab and hyaluronidase-fihj combined with pomalidomide and dexamethasone.

Cardiac monitoring has not been adequately established for such patients.

Florian Thomas, MD, and Adrian Hepner, MD, cover the basics of the degenerative nerve disorder.

The new PDUFA date for avacopan has been pushed back to October 7.

Mortality rates differed significantly across the 3 clusters.

Patients with chronic kidney disease reported poor quality of life, particularly in domains related to their condition.

Strategies to avoid complications of sickle cell disease and ensure non-dependency on opioids when treating patients for pain as a result of the disease.

This past year has brought unique challenges for sickle cell disease patients and communities of color. The ongoing vaccination campaign is yet another challenge that healthcare communities must find ways to overcome.

A sickle cell expert reflects on the state of care and access for sickle cell communities.

Younger individuals and those with specific sickle cell genotypes led to earlier predictions for fetal risk of sickle cell disease.

Older age, macroalbuminaria, and high diastolic blood pressure are among some of the risk factors significantly associated with onset of chronic kidney disease.

More studies are needed to determine whether bi-level positive airway pressure ventilation can help prevent acute chest syndrome.

An expert in hematology/oncology discusses the emerging role of gene therapy to treat sickle cell disease.

Considerations for using HSCT (hematopoietic stem cell transplantation) in patients with sickle cell disease.