News

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,…

Crizanlizumab (SEG101), manufactured by Switzerland’s Novartis, delays the time to first sickle cell pain crisis (SCPC) in adults with sickle cell disease, according to results from a subgroup analysis of the Phase II SUSTAIN study (NCT01895361). Researchers presented the abstract, “Crizanlizumab 5.0 Mg/Kg Increased the Time to First on-Treatment…

The sickle disease of 83 percent of adolescents improved after 16 weeks of taking Global Blood Therapeutics‘ voxelotor, preliminary results of a Phase 2a clinical trial indicate. Another important finding was that voxelotor prevented irregularly shaped oxygen-carrying hemoglobin cells from clumping in more than half of those treated. The clumping prevents…

Prolong Pharmaceuticals has completed enrolling patients in an ongoing Phase 2 clinical trial of  Sanguinate, a treatment for the painful blocked blood vessel episodes that occur in sickle cell disease. The cause of the episodes, known as vaso-occlusive crises, is abnormally shaped red blood cells  bunching up and blocking…