Articles

Podcasts

Videos

Rare Disease Report®

ADM Giroir, MD, discusses additional examples of efforts to optimize sickle cell care.

ADM Brett Giroir, MD: Mobilizing a Response to Sickle Cell

The US Assistant Secretary for Health provides a top-down, government-level perspective on how sickle cell care has advanced in recent years.

ADM Brett Giroir, MD: The Momentum of Sickle Cell Care

The past few years have seen emerging therapies, but the tragedies of 2020 have only amplified the healthcare inequalities associated with sickle cell care.

2020 Highlights the Disparities and Discrepancy of Sickle Cell Care

Newer Treatment Options to Manage Sickle Cell Disease

FLT3-mutated patients achieved favorable antileukemic responses regardless of anthracycline type or gilteritinib dosing regimen. 


Early Results Suggest Gilteritinib Plus Chemotherapy Safe, Effective for AML

Children with sickle cell disease have the opportunity to learn about STEM concepts in the context of their own condition.


Novel Educational Programs Can Benefit and Engage Sickle Cell Patients

Early initiation reduces hospital stays and length of IV opioid use.

Early Initiation of Rivipansel for VOC in Sickle Cell Disease

The furthest-progressed gene-edited therapy for a genetic disease reported historic efficacy data in ongoing phase 1/2 trials.

Gene-Editing Therapy CTX001 Reports Durable Effects for TDT, Sickle Cell Patients

Patients with acute myeloid leukemia or myelodysplastic syndromes are at especially increased risk for increased disease severity and death.  

COVID-19 and Hematological Diseases Linked to High Mortality Rate

Early assessment from an Australian team of investigators is targeting a pathway linked to hemoglobin expression.