News

Bluebird Bio is set to resume trials of LentiGlobin, its investigational gene therapy for sickle cell disease, after the U.S. Food and Drug Administration (FDA) lifted its clinical hold. The company is now working closely with study investigators and clinical trial sites to resume normal activities as soon…

In children with sickle cell disease (SCD), ages 4–11, weight-based doses of Oxbryta (voxelotor) result in similar rises in hemoglobin levels and reductions in red blood cell destruction (hemolysis) to those observed in older patients at the approved dose, six-month data from the ongoing Phase 2 HOPE-KIDS 1…

Those who wish to gain practical tools for living optimally with rare diseases are encouraged to attend the annual Living Rare Living Stronger Patient and Family Forum, hosted by the National Organization for Rare Disorders (NORD) and set this year for June 26-27. The conference brings together patients,…

A Promising Innovative Medicine (PIM) designation has been granted to Oxbryta (voxelotor), a treatment to reduce the destruction of red blood cells in people, 12 and older, with sickle cell disease (SCD). The designation, given to promising therapies that are likely to provide major benefit to patients, follows…

Eurordis opened a campaign, called Rare 2030 Action, that is seeking to establish a European action plan for rare diseases to ensure that none of the 30 million people in Europe living with rare diseases are left behind by the start of a new decade. As part of…

From sporting red gear to entering a spoken word contest, supporters globally are poised to mark World Sickle Cell Awareness Day, an annual event that takes place on June 19. The goal is to heighten awareness of sickle cell disease (SCD), an inherited blood disorder that affects millions of…

Ahead of this year’s Rare Disease Week on Capitol Hill, held virtually July 14–22, the EveryLife Foundation will award grants to top advocates of rare disease organizations who participate in the week’s pre-events. The top 50 point-earners will be eligible to win $1,000 to $5,000in  grants, totaling up…

Machine learning tools can identify sickle cell disease (SCD) patients at high risk of progressive kidney disease as early as six months in advance, a study shows. The study, “Using machine learning to predict rapid decline of kidney function in sickle cell anemia,” was published in the journal…

The Saudi Food and Drug Authority (SFDA), in Saudi Arabia, will review Emmaus Life Sciences’ application for the approval of its oral medication Endari (L-glutamine) for treating sickle cell disease (SCD). Given that the SFDA’s review process typically takes 12 to 18 months, a decision is not expected before…

Rapid and repeated imaging of blood flow through the eyes can inform physicians of a patient’s risk of sustaining eye damage and developing vision problems related to sickle cell disease (SCD), as well as of how effective a given therapy is, a recent study shows. According to researchers, this…