McKesson Plasma and Biologics LLC will distribute Endari, a therapy indicated to reduce the acute complications of sickle cell disease (SCD) in adult and pediatric patients age 5 and older. The announcement was made by the Endari’s developer, Emmaus Life Sciences, Inc.. This distribution agreement enables…
Hoffmann-La Roche has sold the worldwide rights to its discontinued heart disease drug, inclacumab, to Global Blood Therapeutics (GBT) for $2 million and milestone payments. GBT plans to investigate inclacumab as a treatment for painful blockages of blood vessels in patients with sickle cell disease (SCD). The technology transfer process…
Siklos (hydroxyurea 100 mg tablets) — indicated for the treatment of pediatric patients, ages 2 and older, with sickle cell anemia (SCA) — can now be ordered in U.S. pharmacies. Developed by French pharmaceutical company ADDMEDICA, Siklos is now exclusively marketed in the U.S. by Medunik USA…
The National Institutes of Health (NIH) has awarded $4 million to the departments of emergency medicine and hematology at the Icahn School of Medicine at Mount Sinai Hospital in New York City to test a creative new approach for treating sickle cell disease (SCD). The strategy involves inhaled corticosteroids that…
Some treatments for sickle cell disease or cancer can destroy germ cells that later develop into sperm, putting young boys at risk of reduced fertility in adulthood, a study suggests. These results could lead to a change in treatment guidelines, giving healthcare providers who are treating prepubescent boys the…
A U.S. Senate committee unanimously supported the Sickle Cell Disease Act of 2018, and Emmaus Life Sciences is also announcing its support of the bill. The Sickle Cell Disease Research, Surveillance, Prevention and Treatment Act of 2018 (S. 2465) seeks to improve the understanding of the healthcare system regarding sickle…
The oral therapy Endari (L-glutamine), developed by Emmaus Life Sciences, will soon be made available to patients with sickle cell disease in the United States military, along with 9.4 million of their beneficiaries, thanks to a new distribution agreement, the company announced. The agreement is between Emmaus and…
The HRI signaling protein was identified as a key negative regulator of hemoglobin production in red blood cells, unraveling a potential new therapeutic target for sickle cell disease (SCD). Using the gene editing tool CRISPR-Cas9, HRI was found to block the production of fetal hemoglobin, which carries iron…
Treatment with Endari (L-glutamine) leads to fewer pain crises and hospitalizations in patients with sickle cell disease, according to final results of a Phase 3 clinical trial. These results were published in the study, “A Phase 3 Trial of l-Glutamine in Sickle Cell Disease,” in the…
A simple blood test and other measures could help halt the rise of sickle cell anemia in Africa and prevent fatal complications, especially among children, a World Heath Organization (WHO) official said this month. People living in Africa make up some 66 percent of the approximately 120 million people…
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