Naproxcinod, an investigational therapy being developed by Fera Pharmaceuticals as a treatment for sickle cell disease (SCD), has been granted orphan drug designation by the U.S. Food and Drug Administration (FDA). Orphan drug status provides regulatory support and financial benefits to experimental treatments for diseases that affect fewer…
An enzyme known to play a key role in blood vessel health is present at low levels in the cells lining the lung blood vessels of patients with sickle cell disease (SCD), which may affect how these cells stick to their supporting matrix, a study has found. Lack of…
Bionews, the publisher of this website, hosted a virtual panel discussion on Rare Disease Day 2022, taking a deeper dive into what it’s like to live with a rare disease, including conversations about advocacy, mental health, survivor’s guilt, treatment of minority patients, and more. The Monday event, “A…
A team of international researchers has received a $3-million grant from the National Institutes of Health (NIH) to sequence the genome — the whole genetic code — of children with sickle cell disease (SCD) in Ghana. The award was granted to the project’s principal investigator, Solomon Fiifi Ofori-Acquah, PhD,…
A rare disease puts an economic burden on the patients, families, and caregivers that it affects, and will no doubt be an integral part of discussions on Rare Disease Day 2022, which brings international awareness about the more than 300 million people living with rare disorders. Part of that…
Red blood cell biomarkers may predict sickle cell disease (SCD) severity and the risk of vaso-occlusive crisis, according to a study that evaluated the FA-WB-VCAM blood test. “Our latest research indicates that one of our clinically available red blood cell health tests called Flow Adhesion of Whole Blood to…
The nonprofit RARE-X is creating an easily-accessible, centralized data hub for all rare disease patient data that can help researchers answer questions about existing disorders, discover new ones, and work toward finding treatments. It was spun out of the work that Nicole Boice, founder and chief engagement officer of…
Researchers have ruled out a phenomenon called clonal hematopoiesis as the potential cause of the increased risk of blood cancer in people with sickle cell disease (SCD) by analyzing the genetic data of more than 3,000Â SCD patients and 71,000 unaffected people. In clonal hematopoiesis, people acquire mutations that cause…
It’s been nearly a year since the EveryLife Foundation for Rare Diseases released its expansive report finding the total economic burden of rare disorders in the U.S. to be nearly $1 trillion.
Siklos (hydroxyurea) is now approved in the U.S. to reduce the frequency of painful crises and lower the need for blood transfusions in adults with sickle cell anemia, the most common and usually the most severe form of sickle cell disease. The medication already was approved for…
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