RDR Alert®

New study finds that telehealth is a viable option for treating sickle cell anemia patients in rural populations.

Voxelotor has the potential to decrease long-term problems in pediatric sickle cell disease.

Kim Smith-Whitley, MD, discussed her research on voxelotor as treatment for children with sickle cell disease.

Voxelotor study results show it’s just as effective in children as adults and adolescents.

Obstructive sleep apneas and overnight hypoxemia are more likely to develop in patients with sickle cell disease.

The Firmicutes/Bacteroidetes ratio was lower for patients with sickle cell disease compared to a healthy control group.

Ketamine was not superior to morphine in reducing pain scores.

The main mechanism for exercise intolerance is impaired blood oxygen carrying capacity.

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

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.

The rare liver disease can often lead to liver failure.

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.

Patients with glucocorticoid-refractory or -dependent chronic GVHD are more likely to experience complete or partial response with the JAK inhibitor than with control therapies.

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

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.

An expert ophthalmologist offers lessons and insights into the rare genetic eye disorder.

Mortality rates differed significantly across the 3 clusters.