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…
News
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 3,000 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…
Oxbryta (voxelotor) has become the first approved therapy in the European Union to target the underlying cause of sickle cell disease (SCD) for patients 12 and older. The medication, given as a once-daily oral tablet, suppresses the sickling and destruction of red blood cells that underlies the disease. Eligible…
The supplemental insurance provider Aflac is expanding its “My Special Aflac Duck” social robot program to benefit children with sickle cell disease (SCD). Healthcare professionals and nonprofit organizations can order the life-sized robotic companion for free for children ages 3 and up who have cancer or SCD.
A lung ultrasound can aid in accurately diagnosing a serious respiratory complication called acute chest syndrome in children with sickle cell disease (SCD), a study reported. The study, “Point-of-care lung ultrasound is more reliable than chest X-ray for ruling out acute chest syndrome in sickle cell…
Since 2008, Rare Disease Day — the last day of February — has brought together patients, caregivers, family members, friends, and advocates from around the world to raise awareness and improve equity for the more than 7,000 known rare diseases that affect more than 300 million people. In 2022, the…
A researcher will use a four-year, $1.63 million grant from the National Institutes of Health (NIH) to help set up a quality control system for CRISPR-based therapies aimed at certain genetic disorders, starting with sickle cell disease (SCD). The grant was awarded to Kiana Aran, PhD, an associate…
Adakveo (crizanlizumab), by Novartis, is now available on the U.K.’s National Health Service (NHS) for sickle cell disease (SCD) patients 16 and older. The new treatment, which is delivered by an into-the-vein infusion, can be used alone or as an add-on to hydroxyurea to help reduce the frequency of…
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