Sickle Cell

Patients with Fabry disease who received treatment with PRX-102 every 4 weeks displayed a stable condition at the end of the trial.

Novartis announces promising results from research evaluating the safety and efficacy of Zolgensma.

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.

Why is Rare Disease Day so important? Hear the answers from some of the most qualified professionals in rare disease fields.

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.

An overview on the pathophysiology of sickle cell disease and expert perspectives on the clinical (and psychosocial) burden it presents.

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.

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

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.

While Omicron cases surge, Valentina, Baez-Sosa, MD, speaks on the national blood shortage and the risk it poses to patients with sickle cell disease in particular.

Maureen Achebe goes into detail about the open label extension of the HOPE trial that evaluated to use of Voxelotor in patients with sickle cell disease and anemia.

As the year begins, Edward Ivy addresses what's currently happening in the field of sickle cell disease from the impact of the blood shortage to exciting new therapies.

Alloimmunization is more prevalent among patients with sickle cell disease than previous literature states. Baez-Sosa discusses her study and treatment practices.

After conducting and extensive literary review on all eligible data regarding COVID-19 in patients with sickle cell disease or trait, experts discuss what is known and what needs to be better understood about the pandemic's effect on this population.

Investigators conducted an extensive literature review on clinical COVID-19 outcomes in patients with SCD or SCT to better navigate treatment during the pandemic.

Regina Hartfield is the new CEO and president of the Sickle Cell Association of America. Listen to our interview with her to find out what's in store for 2022.

Baez-Sosa explains why further investigation of alloimmunization in patients with sickle cell disease is crucial.

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.