Sickle Cell

Reflections on optimal approaches to patient follow-up, particularly regarding pain management and inherent risks in high-dose opioids.

A focused discussion on how comprehensive care is applied by multidisciplinary teams to improve the management of sickle cell disease.

The clinical trial of efanesoctocog alfa provided positive top-line results for treating patients with severe Hemophilia

Investigators use an indirect approach to estimate the mortality rate of children with sickle cell anemia in sub-Saharan Africa because there's no systematic screening at birth.

A broad perspective on the value of multimodality care in the management of patients diagnosed with sickle cell disease.

Wally Smith, MD, and Payal Desai, MD, spearhead a discussion on patient education surrounding sickle cell disease symptomology and lifestyle modifications.

The FDA granted accelerated approval to the treatment of adults with intermediate or high-risk primary or second myelofibrosis.

Expert Mikkael Sekeres, MD, explains that the COVID-19 pandemic affected all of medicine, but in a rare disease domain that impact was even greater.

The president of Make-A-Wish New Jersey speaks about the impact of granting a wish to a child living with a rare disease.

Expert perspectives on the importance of newborn screening to impact mortality rates in sickle cell disease.

Expert panelists reflect on the global prevalence of sickle cell disease, as well as general disease awareness.

Shared insight on how a patient’s life may progress following a diagnosis of sickle cell disease, along with considerations for pediatric versus adult management.

Rare disease pyruvate kinase deficiency received first approved disease-modifying therapy.

Progressive familial intrahepatic cholestasis is a genetic liver disease that doesn't only affect the children who are diagnosed, but also the caregivers responsible for them.

Dr. Osunkwo details advancements in the field of sickle cell disease, including chronic disease-managing therapies and stem cell transplant and gene therapies.

Investigators developed a new method to identify potential treatments for hundreds of diseases by profiling drugs approved by the FDA.

The renowned expert joins to discuss the clinical characteristics, societal burdens, and promising therapy research surrounding the hematologic disease.

The monoclonal antibody is designed to reduce the need for red blood cell transfusions to hemolysis among adults with the rare disease.

The recombinant von Willebrand factor, VONVENDI, receives approval from the FDA to treat patients living with severe type 3 von Willebrand disease.

In the midst of a national blood crisis, the FDA is funding a study to examine if different questioning can be used to determine the HIV risk of gay or bisexual men who want to donate blood.

Dr. Ivy speaks on promising new therapies and training for sickle cell management during the first blood shortage in the United States in 10 years.

Exciting new therapies for sickle cell disease are on the horizon. We explored some of the most promising investigational therapies in recognition of National Blood Donor Month.

Achebe explains the promising results of the open label extension to the HOPE trial for patients with sickle cell disease.

Phase 3 trial examines if adjusting the dose of hydroxyurea reduces the risk of stroke in children with sickle cell anemia.

Accurate results can be obtained rapidly with this intuitive whole blood hemostasis system, allowing for efficient workflow at the point of care.