News

An abundance of events are afoot around the world to mark Rare Disease Day 2020 on Feb. 29. The activities are focused on heightening awareness about rare diseases and the hundreds of millions of individuals they are thought to affect. Patients, caregivers, and advocates worldwide will sport denim ribbons…

African-Americans with sickle cell trait and sickle cell disease have a significantly faster decline in kidney function than their unaffected peers with normal hemoglobin, a study suggests. Men, people with diabetes, and those with good kidney function to start saw kidney function decline fastest, according to…

The Howard University College of Medicine has been awarded a $42,000 grant to support its “LIFE (Learning Is Fundamental for Everyone) with Sickle Cell Disease” project for youth with the inherited blood disorder and their families. The grant, from the Walter Brownley Trust, was awarded to the College of Medicine’s…

Pediatric patients with sickle cell disease complicated by acute kidney injury (AKI) were more likely to be admitted to the intensive care unit (ICU), had longer hospital stays, accumulated greater hospitalization costs, and had a higher rate of readmission, a large-scale analysis…

The U.S. Food and Drug Administration (FDA), a vast government bureaucracy, employs about 17,500 people and had a budget of $5.7 billion in 2019. Yet even with its enormous resources, the FDA these days relies more and more on patients to…

More than a quarter of patients with sickle cell disease (SCD) are readmitted to the hospital within 30 days of being discharged, most often due to sickle cell crisis, according to a recent U.S. nationwide study. This high rate of readmissions was associated with a significant financial burden…

The gleaming new Dutch headquarters of the European Medicines Agency (EMA), fronting Domenico Scarlattilaan in Amsterdam’s suburban Zuidas business district, finally opened for business last month — just over two years after the European Union decided to relocate the EMA to the Netherlands in the wake of Brexit.

A recent survey found that U.S. residents are largely interested in and support continued research into the role of human genetics in treating and possibly curing illnesses such as sickle cell disease (SCD). Commissioned by the American Society of Human Genetics (ASHG) in partnership with Research!America, the…

People with sickle cell disease (SCD) treated with hydroxyurea in the U.S. still face many challenges, including the financial burden posed by their healthcare expenses, low treatment adherence and high treatment discontinuation rates, according to a study based on pharmacy claims data. The study, “Treatment patterns and…

FDA-approved therapies for sickle cell disease (SCD) may not be cost-effective at their current prices, according to a draft report issued by the Institute for Clinical and Economic Review (ICER). The report focused on the clinical benefits and cost-effectiveness of Novartis’ Adakveo (crizanlizumab), Global…