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Ennis James, MD, discusses the challenges and lingering questions associated with sarcoidosis 

The Rare Disease Report Podcast: Sarcoidosis

Elevated B cell activating factor levels in children and adults were associated with the development of FVIII inhibitors, which may lead to FVIII infusion failures.

Study Identifies Cytokine Target for Treatment-Resistant Hemophilia A

SDOCT imaging shows that sickle cell patients with no clinical evidence of retinopathy have considerably lower central macular thickness when compared with healthy controls. 

Sickle Cell Anemia Linked to Significant Retinal Thinning

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

Study Describes Nutritional, Growth Deficits in Pediatric Sickle Cell Patients

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


Long-Term HOPE Data Supports Efficacy, Safety of Voxelotor for Sickle Cell

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

Hydroxyurea for Sickle Cell Anemia Linked to Improved Academic Performance 

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

FDA Approves Alirocumab for Homozygous Familial Hypercholesterolemia

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

Sotatercept Could Be a Promising Novel Agent for Pulmonary Arterial Hypertension

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

FDA Expands Daunorubicin and Cytarabine Label for Pediatric AML Patients

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

Sickle Cell Gene Therapy Could Significantly Impact Medicaid Budgeting, Access

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