News

Blood drive, walks planned as part of Sickle Cell Awareness Month

Fundraising walks, a golf tournament, and a virtual blood drive are among activities planned to raise awareness of sickle cell disease (SCD) in September, National Sickle Cell Awareness Month. The campaign aims to raise funding for research into the inherited blood disorder and to support the approximately 100,000 U.S. residents…

Inclacumab fails to reduce pain crises vs. a placebo in SCD trial

Pfizer’s investigational therapy inclacumab failed to outperform a placebo in reducing the rate of vaso-occlusive crises — episodes marked by painful inflammatory responses — in people with sickle cell disease (SCD), according to new data from the Phase 3 THRIVE-131 study. “We recognize this news is disappointing…

Gene-edited cell therapy for severe SCD lands new FDA designation

The U.S. Food and Drug Administration (FDA) has granted regenerative medicine advanced therapy (RMAT) designation to Beam Therapeutic’s BEAM-101, a one-time, gene-edited cell therapy for severe sickle cell disease (SCD). RMAT status is granted to therapies that have shown potential for treating serious or life-threatening conditions. It provides a…

Sleep issues linked to greater healthcare needs in SCD children

Children with sickle cell disease (SCD) who have sleep problems, such as sustained low blood oxygen during sleep, or nocturnal hypoxemia, require more healthcare resources than children with normal sleep parameters, a study reports. “These findings suggest that targeting nocturnal hypoxemia and related sleep disturbances … through appropriate screening…