News

The Children’s National Health System hospital in Washington has recruited two of the country’s leading hematology and sickle cell disease experts to lead its cutting-edge blood disorders program. Dr Suvankar Majumdar will head the hospital’s Division of Hematology, one of the country’s largest and most comprehensive blood disorders programs. It is…

The U.S. Food and Drug Administration (FDA) approved Imbruvica (ibrutinib) for treatment of adult patients with chronic graft-versus-host-disease (cGVHD) who failed to respond to other forms of systemic therapy, AbbVie announced. FDA’s decision means Imbruvica is the first and only therapy specifically approved for adults with cGVHD, a serious outcome that…

Sancilio Pharmaceuticals has completed enrolling patients in a Phase 2 clinical trial evaluating Altemia (docosahexaenoic acid) as a treatment for children with sickle cell anemia (SCA) and sickle cell disease (SCD). The SCOT trial (NCT02973360) is aimed at finding an optimal dose of the therapy as well as…

The Israeli government has awarded Gamida Cell a $3.5 million grant to advance the development of clinical trials for CordIn, a therapy to treat sickle cell disease (SCD) and thalassemia. The money, granted by the Ministry of Economy and Industry’s Israel Innovation Authority (IIA), will also support further development and sales of…

Introducing a “natural” genetic mutation into the blood cells of people with sickle cell and like diseases, using CRISPR  gene editing, was seen to restore the production of fetal hemoglobin and offer a way to treat these disorders, researchers report. Their study was published in the journal Blood, in an article titled “KLF1 drives the expression…

Sema4, an interdisciplinary health information company, has partnered with Helix, a personal genomics company, to launch CarrierCheck — a simple, saliva-based test that screens for 67 conditions, including sickle cell disease (SCD). The test may provide individuals with a snapshot of how their DNA may affect their future…

Couples who wish to know the risk of passing on a genetic disease such as sickle cell anemia to their children may now do so with QHerit, a new screening test by Quest Diagnostics. The QHerit Pan-Ethnic Expanded Carrier Screen test covers 22 genetic diseases and was produced…

Doctors treating pediatric patients with sickle cell disease (SCD) for pain associated with a vaso-occlusive crisis (VOC) often have few optimal therapy options. But a recent study reviewed a technique that shows potential for mitigating pain in child SCD patients who are experiencing VOC.

Global Blood Therapeutics has expanded its ongoing HOPE-KIDS 1 trial (NCT02850406), evaluating the investigative GBT440 in pediatric patients with sickle cell disease, to include a new single-dose cohort in younger children ages 6 to 11. GBT440 is being developed as an oral, once-daily drug candidate for the treatment…

Endari (L-glutamine) today became the first therapy in almost 20 years to win approval from the U.S. Food and Drug Administration to treat sickle cell disease. In giving the green light to Endari use in patients starting at age 5, the FDA also made the oral drug the first ever…