News

Nominations are now open for the worldwide 2022 Black Pearl Awards from Eurordis-Rare Diseases Europe. The 12 award categories recognize individual advocates, policy makers, researchers, organizations, and companies who work to make a difference for the global rare disease community. The deadline for nominations is Sept. 10…

As a stem cell transplant is the only curative treatment for sickle cell disease (SCD), a blood disorder that disproportionately affects Blacks and African Americans, “Cheek Week” is again calling for the participation and inclusion of donors from different ethnic backgrounds in the Be The Match registry,…

A new research collaboration between Jasper Therapeutics and Aruvant Sciences will evaluate the use of JSP191, Jasper’s anti-CD117 monoclonal antibody, as a conditioning agent for ARU-1801, Aruvant’s experimental gene therapy for sickle cell disease (SCD). “The unique attributes of ARU-1801 enable us to bring a potentially curative one-time therapy…

A single dose of CTX001, an experimental gene-editing cell therapy, rapidly increases the levels of hemoglobin and prevents vaso-occlusive crises (VOCs) for up to nearly two years in people with severe sickle cell disease (SCD), according to interim data from the Phase 1/2 CLIMB-SCD-121 clinical trial.

Note: This story was updated July 7, 2021, to clarify that Hertz Nazaire lived with his godmother, rather than his grandmother, after his mother’s death.  A Connecticut-based artist living with sickle cell disease and blindness in his right eye is using his art to advocate for…

Velvet Brown-Watts was devastated when her son, Jeremiah Watts Jr., now 16, was diagnosed with sickle cell disease in 2004 at 2 months old. “It was probably one of the worst things I ever heard,” Brown-Watts, 46, told Sickle Cell Disease News. Doctors at…

At its first virtual investor event, biotech company Centogene set a bold mission: to cure 100 rare diseases within the next decade. A leader in the field of genetic diagnostics, Centogene used the June 22 event to present its strategic priorities, outlining its plans to speed the discovery…

To improve life for people with sickle cell disease (SCD), Global Blood Therapeutics (GBT) has awarded grants worth $50,000 each to nine community-based, nonprofit groups and institutions across the U.S. to speed the development and use of sustainable, access-to-care programs. These one-year grants are part of GBT’s Access to…

Global Blood Therapeutics (GBT) is currently focused on expanding the use of Oxbryta (voxelotor) to children, ages 4–11, with sickle cell disease (SCD). But research into its use with younger children, as well as studies into other potential SCD therapies, are also underway. “Sickle cell disease patients of all ages worldwide…

To help patient advocacy leaders and their partners better understand how global health statistics codes — known as ICD codes — are assigned, updated, and revised in the U.S. health information system, the EveryLife Foundation for Rare Diseases is presenting a first-of-its-kind resource guide. The foundation created the…