ARU-1801, Aruvant Sciences’ experimental gene therapy for sickle cell disease (SCD), led to the production of fetal hemoglobin in red blood cells and significantly lowered the number of vaso-occlusive crises (VOCs) in a group of five people with severe SCD, according to data from the ongoing Phase…
News
CRISPR Therapeutics and Vertex have launched two Phase 3 trials to assess the safety and effectiveness of CTX001, an experimental gene-editing cell therapy, one in children with sickle cell disease (SCD) and another for those with transfusion-dependent beta thalassemia (TDT). Both Phase 3 studies will enroll up to…
Hemex Health’s portable point-of-care device, Gazelle, is being enhanced to more precisely monitor fetal hemoglobin levels. According to Hemex, this could be useful for monitoring people with sickle cell disease (SCD) who are being treated with hydroxyurea. The company announced the software upgrade on May 8…
Graphite Bio’s GPH101, an investigational gene-editing therapy that aims to correct the genetic mutation that causes sickle cell disease (SCD) and potentially cure the condition, has been awarded fast track designation by the U.S. Food and Drug Administration (FDA). The fast track process is designed to accelerate the…
A Russian military plane crash near Tetiana Zamorska’s home in Kyiv, Ukraine, was a sign that it was time for her and her family to leave. The treacherous, 34-hour pilgrimage that ultimately brought the group of eight by car to temporary accommodations in neighboring Poland last month was physically and emotionally difficult,…
Robotic ducks were delivered recently as comfort companions to children with sickle cell disease (SCD) at the Children’s Regional Hospital at Cooper in South Jersey. The life-sized cuddly robot, called “My Special Aflac Duck,” were made available free to children, 3 and older, in a new collaboration between…
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…
Recent Posts
- Agios to seek accelerated approval in US of oral mitapivat for SCD
- Japanese agency awards $32M to advance sickle cell treatment to trials
- Intentional lifestyle changes got me through winter without a crisis
- Chicago nurses lead charge to speed up SCD emergency care: Study
- Recognizing the warning signs and symptoms of a sickle cell crisis
- US senators seek 5 more years of federal funding for stem cell program
- Adults with SCD face high risk of stroke in their 30s and 40s, study finds
- Sickle cell doctors face higher burnout than other specialists: Study
- Disease burden heavy for SCD patients in US, despite promise of gene therapy
- The conversation we avoid: Telling children they have sickle cell disease