Marisa Wexler, MS,  senior science writer—

The U.S. Food and Drug Administration (FDA) has granted both orphan drug and rare pediatric disease designations to inclacumab and GBT601, two experimental therapies for sickle cell disease (SCD) being developed by Global Blood Therapeutics (GBT). These designations confer benefits to therapy developers that are designed to speed…

Hemex Health’s portable point-of-care device, Gazelle, is being enhanced to more precisely monitor fetal hemoglobin levels. According to Hemex, this could be useful for monitoring people with sickle cell disease (SCD) who are being treated with hydroxyurea. The company announced the software upgrade on May 8…

The European Commission is expected to propose a new governing framework for health data next month, called the European Health Data Space (EHDS), with the aim of connecting national health systems to facilitate secure and efficient transfer of data across systems in different European nations. The move is expected to…

Endari, an oral therapy approved to treat sickle cell disease (SCD) has been added to the Florida Medicaid Preferred Drug List (PDL), its manufacturer Emmaus Life Sciences announced. The PDL, according to the Florida Agency for Health Care Administration (AHCA), is a listing of medications that are considered…

Siklos (hydroxyurea) is now approved in the U.S. to reduce the frequency of painful crises and lower the need for blood transfusions in adults with sickle cell anemia, the most common and usually the most severe form of sickle cell disease. The medication already was approved for…

A lung ultrasound can aid in accurately diagnosing a serious respiratory complication called acute chest syndrome in children with sickle cell disease (SCD), a study reported. The study, “Point-of-care lung ultrasound is more reliable than chest X-ray for ruling out acute chest syndrome in sickle cell…

Most children with sickle cell anemia (SCA), the most common and often the most severe form of sickle cell disease (SCD), do not receive preventive antibiotic treatment or participate in yearly stroke screenings that are recommended for those with the condition, according to a study based on U.S. data.

A new formulation of Oxbryta (voxelotor) that is suitable for treating children as young as 4 is now available in the U.S. at specialty pharmacies. Late last year, the U.S. Food and Drug Administration (FDA) expanded the approval of Oxbryta to treat children with sickle cell disease (SCD)…

Agios Pharmaceuticals has launched a Phase 2/3 trial called RISE UP to test its investigational therapy mitapivat (AG-348) in people with sickle cell disease (SCD). The trial (NCT05031780), which is not yet recruiting, aims to enroll about 237 people with SCD, ages 16 and older. To be…

The first participant has been enrolled in the Phase 1/2 clinical trial CEDAR evaluating Graphite Bio’s investigational gene editing therapy GPH101, which is designed to directly correct the genetic mutation that causes sickle cell disease (SCD). “We are thrilled that our first patient is now enrolled in our CEDAR…