News

Two kinds of CRISPR gene editing therapies show promise as treatments for a number of blood diseases, including sickle cell anemia, Editas Medicine reports. Gene editing is one of a wave of new approaches to treating diseases. It involves using specialized proteins to make precise cuts and pastes of DNA to correct…

Researchers found that treatment with hydroxyurea at currently approved doses for sickle cell anemia leads to a significant drop in total sperm count in men. The study, “Adverse effect of hydroxyurea on spermatogenesis in patients with sickle cell anemia after six months of treatment,” was published in the…

Emmaus Life Sciences has reached an agreement with the Centers for Medicare and Medicaid Services that will allow coverage of Endari (L-glutamine oral powder) to reduce acute complications of sickle cell disease (SCD) in adults and children 5 years and older. “This treatment is considered very important by many…

This week marks the launch of the “7,000 Mile Rare Movement,” a nationwide effort to raise money for research into the 7,000 known rare diseases that afflict at least 30 million Americans. The campaign kicks off Feb. 1 and culminates with Rare Disease Day on Feb. 28. Organized by…

A man and woman who are both carriers of the sickle cell anemia mutation can have a healthy child by fertilizing eggs in a lab, then deciding which of multiple embryos that develop from the eggs will become the baby, a case study indicates. It also showed that umbilical cord…

England’s National Health Service (NHS) has selected 138 entrepreneurs to design and deliver new technological solutions for healthcare challenges – one of which was appointed to tackle conditions like sickle cell disease (SCD) using a voice-recognition mobile app. The support is part of the NHS Clinical Entrepreneurs…

The U.S. Food and Drug Administration (FDA) approved Endari (L-glutamine) last summer, but now the medicine is available by prescription to most patients in the U.S. who have sickle cell disease. Endari, an oral L-glutamine therapy, was developed by Emmaus Life Sciences to reduce acute complications of…

Voxelotor, a potential treatment for sickle cell anemia (SCA), has been given breakthrough therapy designation (BTD), Global Blood Therapeutics (GBT) recently announced. Voxelotor (previously called GBT440) is a potential once-daily oral medicine for SCA patients. It was designed to increase hemoglobin’s binding of oxygen, which keeps red blood…

Emmaus Life Sciences has developed a sickle cell disease therapy that the United States has approved but regulators in much of the world are still reviewing. Until other countries approve L-glutamine, those living there will be unable to obtain the treatment for the blood disease, which is painful, causes organ damage…

Bluebird bio announced encouraging interim results from the ongoing Phase 1 clinical trial testing its investigational gene therapy LentiGlobin for patients with severe sickle cell disease (SCD). The results were presented at the 59th Annual Meeting of the American Society of Hematology (ASH), held Dec. 9-12 in Atlanta,…