Category: News

CONSA Update: “Newborn screening for sickle cell disease in Africa” Article

The Consortium of Newborn Screening in Africa (CONSA), launched in 2016 to address the global burden of SCD, shows the value of newborn screening and how it can be implemented in diverse settings throughout sub-Saharan Africa. This American Society of Hematology (ASH) supported consortium aims to introduce standard-of-care practices for screening and early-intervention therapies at participating …

Advocates Lobby US Congress During Virtual Rare Disease Week

More than 600 people participated in the 10th annual Rare Disease Week on Capitol Hill 2021, held virtually July 14–22, to advocate for the rare disease community. Hosted by the EveryLife Foundation’s Rare Disease Legislative Advocates (RDLA) program, the event brings together community members from across the U.S. to learn about federal legislative issues, meet other …

Sickle cell disease and a pivotal moment to end health inequality

Covid-19 laid bare the long-standing vulnerability of minority and low-income communities in U.S. society and its health system even as we celebrated the power of scientific innovation to rein in the pandemic. It’s time to turn that innovation, driven by an even greater mind shift, to end inequality in treatment. In perhaps no condition is …

SCDC Released New Fact Sheets on Crizanlizumab, L-glutamine, & Voxelotor

The Sickle Cell Disease Coalition (SCDC) recently released SCD Therapy Fact Sheets to offer high-quality, vetted information on SCD treatments approved for use in the U.S. by the U.S. Food & Drug Administration (FDA): Crizanlizumab, L-glutamine, and Voxelotor. The fact sheets are written at a secondary reading level and are designed to help people living with SCD …

Pain Narratives in Children with Sickle Cell Disease (SCD)

This study aims to assess wellbeing (i.e., pain, anxiety, distress) in children and young adults who have, and have not, undergone a transplant. We are recruiting individuals with SCD ages 8-25, and their parent/caregiver from across Canada to partake. This study has been designed to be completed online to ensure convenience and ease for all …

Advancing Treatments and Cures for Sickle Cell Disease

The NHLBI launched the Cure Sickle Cell Initiative in 2018 to support the development of gene-based cures for sickle cell disease (SCD) that will work for all patients, including those ineligible for bone marrow transplants. Researchers involved in the Cure Sickle Cell Initiative helped develop an accurate, affordable, and easy-to-use device for point-of-care diagnosis of SCD in …

CDC’s Sickle Cell Data Collection Program Expands to Nine States

The Centers for Disease Control and Prevention’s (CDC) Sickle Cell Data Collection program gathers health information from multiple sources to determine how many people live with the disease and monitor changes related to their health over time.  In 2020, CDC funded the expansion of the program from two to nine states to collect essential data …

MARAC Statement: Temporary Suspension of Clinical Trials

March 1, 2021 – The Sickle Cell Disease Association of America’s Medical and Research Advisory Committee (MARAC) is aware of the announcement on February 16, regarding the temporary suspension of bluebird bio clinical trials of LentiGlobin Gene Therapy for Sickle Cell Disease and the pause of all commercial use of bluebird bio European gene therapy. …