Articles

Podcasts

Videos

Rare Disease Report®

The Rare Disease Report page is a resource for medical news and expert insights on rare diseases. This page features expert-led coverage, articles, videos and research on the therapies and development of treatments for sickle cell disease, multiple myeloma, and more.

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

Mitapivat Receives FDA Approval for Hemolytic Anemia from Pyruvate Kinase Deficiency

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.

New Study Quantifies the Impact of Caring for a Child with Rare Disease PFIC

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

The Cultural and Clinical Challenges of Sickle Cell with Ifeyinwa “Ify” Osunkwo, MD

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

Investigators Profile FDA Drugs for Modulators of Nonsense-Mediated mRNA Decay

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

The Sickle Cell Disease Cycle with Ify Osunkwo, MD

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

FDA Approves Sutimlimab as First Drug for Cold Agglutinin Disease

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

FDA Approves VONVENDI for Prophylactic Treatment in Adult Patients with VWD

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.

The ADVANCE Study: Rethinking HIV Risk-Assessment for Gay Blood Donors

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. 

Valentina Baez-Sosa, MD: The Blood Crisis is a Huge Concern

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. 

Maureen Achebe, MD, MPH, Shares Extended Durable Data on Voxelotor for SCD

Sickle Cell condition center page, resources on the topics of medical news and expert insight into sickle cell disease can be found. Content includes articles, interviews, videos, podcasts, and breaking news on sickle cell research, treatment, and drug development.

Sickle Cell

Latest News