News

EveryLife Introduces First of Kind ‘Roadmap’ to ICD Codes

To help patient advocacy leaders and their partners better understand how global health statistics codes — known as ICD codes — are assigned, updated, and revised in the U.S. health information system, the EveryLife Foundation for Rare Diseases is presenting a first-of-its-kind resource guide. The foundation created the…

Bluebird to Resume LentiGlobin Trials After FDA Lifts Hold

Bluebird Bio is set to resume trials of LentiGlobin, its investigational gene therapy for sickle cell disease, after the U.S. Food and Drug Administration (FDA) lifted its clinical hold. The company is now working closely with study investigators and clinical trial sites to resume normal activities as soon…

Oxbryta Given Promising Innovative Medicine Status in UK

A Promising Innovative Medicine (PIM) designation has been granted to Oxbryta (voxelotor), a treatment to reduce the destruction of red blood cells in people, 12 and older, with sickle cell disease (SCD). The designation, given to promising therapies that are likely to provide major benefit to patients, follows…

Machine Learning Can Predict Rapid Kidney Function Decline

Machine learning tools can identify sickle cell disease (SCD) patients at high risk of progressive kidney disease as early as six months in advance, a study shows. The study, “Using machine learning to predict rapid decline of kidney function in sickle cell anemia,” was published in the journal…