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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.

Beach shares about Haywood's life, research, and impact on the sickle cell disease community after working alongside him at Johns Hopkins School of Medicine.

Mary Catherine Beach, MD, Honors her Friend, Carlton Haywood Jr, PhD

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

Maureen Achebe, MD, MPH: Sustaining Hemoglobin Levels with Voxelotor

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

Hydroxyurea Examined for Stroke Prevention in Pediatric 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. 

GEM Hemochron 100 Whole Blood Hemostasis Testing System Cleared by FDA

In this interview, Valton details the editing function of TALGlobin01 and how it stands out among traditional gene therapy approaches to sickle cell disease.

Julien Valton, PhD: Restoring the Production of Hemoglobin in SCD Patients 

Björn Mellgård gets technical as he describes the function of TAK-755, the recombinant ADAMTS13 enzyme, in sickle cell disease and cTTP.

Björn Mellgård, MD, PhD: The First Clinical Study of TAK-755 in SCD Patients 

Investigators developed the first module to confront implicit bias toward patients with sickle cell disease on an individual or personal level.

CBT Strategies Help Resolve Implicit Bias Related to Sickle Cell Patients

The American Society of Hematology debuted its Health Equity Rounds series, which are interactive, case-based discussions with an interdisciplinary panel to include hematologists, health equity/public health experts, patients, and patient advocates.

Advocacy: The Solution to Sickle Cell Disease

The clinical program has been suspended for patients that are under the age of 18 after an adolescent patient developed persistent anemia.

FDA Places Partial Hold on Bluebird Bio Sickle Cell Gene Therapy Program

Fuad El Rassi talks about the Grady Comprehensive Sickle Cell center, the COVID-19 vaccine, and the data he presented at ASH. 

Fuad El Rassi, MD: Sickle Cell Disease and COVID-19

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.