Exposure to systemic steroids — frequently used to treat asthma or ease inflammation — is significantly associated with a higher risk of painful vaso-occlusive crises (VOCs) leading to hospitalization in children and adults with sickle cell disease (SCD), a French study shows. “Individuals living with SCD often suffer…
While most adolescent boys and young men with sickle cell disease (SCD) report wanting biological children in the future, few are aware of the impact their disease and its treatments can have on their fertility, a small, pilot U.S. study suggests. Similar knowledge gaps were found for these patients’…
A team of researchers has created the most comprehensive atlas, or roadmap, to date of each step of human blood stem cell development — helping them identify the previously unknown molecular signature of blood stem cells and ending a longstanding debate on these cells’ specific cellular origin. The data, open…
The European Commission is expected to propose a new governing framework for health data next month, called the European Health Data Space (EHDS), with the aim of connecting national health systems to facilitate secure and efficient transfer of data across systems in different European nations. The move is expected to…
Disrupting a specific area of the gene that encodes for adult hemoglobin — which is faulty in people with sickle cell disease (SCD) — led to an increase in the production of fetal hemoglobin, a version of the protein normally produced during fetal development, which can help relieve disease…
A renowned stem cell biologist has been honored with the 2022 Canada Gairdner International Award for his work that set the grounds for a new approach to treating sickle cell disease (SCD) and beta-thalassemia. Stuart H. Orkin, MD, is the David G. Nathan Distinguished Professor of Pediatrics at…
The National Organization for Rare Disorders (NORD) has updated its State Report Card to make it more digitally friendly and added telehealth to its categories of rare disease policy issues in a nod to its increased use during the ongoing COVID-19 pandemic. NORD’s report card project began seven…
Emmaus Life Sciences has launched a full-service telehealth platform that will allow people in the U.S. with sickle cell disease (SCD) greater and easier access to the approved therapy Endari (L-glutamine). Patients will be able to receive a same-day physician’s prescription for Endari, which can…
Imara is discontinuing the clinical development of tovinontrine (IMR-687), its experimental oral therapy for sickle cell disease (SCD) and beta-thalassemia, based on interim data from two Phase 2b clinical trials. While the therapy was generally safe and well tolerated in adults with both inherited blood disorders, it resulted in…
Endari, an oral therapy approved to treat sickle cell disease (SCD) has been added to the Florida Medicaid Preferred Drug List (PDL), its manufacturer Emmaus Life Sciences announced. The PDL, according to the Florida Agency for Health Care Administration (AHCA), is a listing of medications that are considered…
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