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UrduA new study led by Indiana University School of Medicine researchers found a brain network condition called “explosive synchronization” could be the cause of extreme pain crises in people with sickle cell disease.
Tag: Sickle Cell Disease
Children With Sickle Cell Disease Appear to Suffer Eye Complications at Same Rate as Adults
Largest study of children with sickle cell disease finds one in three had retinopathy.
Third Elaine Redding Brinster Prize Awarded for Development of Sickle Cell Disease Therapy
For his work discovering the basis for hemoglobin gene switching and applying those insights to develop a therapy for sickle cell disease and other blood diseases, the Institute for Regenerative Medicine at the University of Pennsylvania awarded Stuart Orkin, MD the third Elaine Redding Brinster Prize in Science or Medicine.
Sickle Cell Disease Continues to Face Underfunding, Lack of Research
Over the years, dedicated sickle cell disease programs and research initiatives have greatly improved patient care and life expectancy. But, giving these patients the care they require still presents a number of challenges, including inadequate funding for sickle cell programs, lack of research, and limited access to healthcare.
News Tip: September Is Sickle Cell Awareness Month — Johns Hopkins Medicine Experts Are Available for Interviews
September is National Sickle Cell Awareness Month. Johns Hopkins Medicine experts who specialize in sickle cell disease are available to speak with reporters about health equity issues related to sickle cell disease.
CHOP and Penn Medicine Researchers Develop “In Vivo” RNA-based Gene Editing Model for Blood Disorders
In a step forward in the development of genetic medicines, researchers at Children’s Hospital of Philadelphia (CHOP) and the Perelman School of Medicine at the University of Pennsylvania have developed a proof-of-concept model for delivering gene editing tools to treat blood disorders, allowing for the modification of diseased blood cells directly within the body. If translated into the clinic, this approach could expand access and reduce the cost of gene therapies for blood disorders, many of which currently require patients receive chemotherapy and a stem cell transplant. The findings were published today in the journal Science.
Base editing shows potential superiority for curing sickle cell disease
Gene therapy that alters hemoglobin genes may be an answer to curing sickle cell disease and beta thalassemia. Scientists at St. Jude Children’s Research Hospital and the Broad Institute of MIT and Harvard found base editing increased fetal hemoglobin production in a new treatment.
Portable Tool to Diagnose and Monitor Sickle Cell Disease Receives U.S. Patent
A new tool will enable patients with sickle cell disease to reliably and conveniently monitor their disease in the same way patients with diabetes can monitor their disease using a glucometer. The goal of managing this inherited, lifelong blood disorder is to prevent acute, painful crises due to sickling and unsickling of red blood cells.
Novel Gene Therapy Proving Safe and Successful in Sickle Cell Patients Treated at Cleveland Clinic Children’s
Researchers presenting preliminary data from a clinical trial aimed at discovering a cure for sickle cell disease reveal positive results among its first patients. Sickle cell disease, a genetic blood disorder, is a painful and debilitating condition for which there are few approved therapies.
St. Jude finds NLRP12 as a new drug target for infection, inflammation and hemolytic diseases
Scientists at St. Jude Children’s Research Hospital found key “on” switch, NLRP12, for innate immune cell death in diseases that cause red blood cells to rupture, which can lead to inflammation and multi-organ failure.
Treatment cost analysis highlights systemic health inequities faced by persons with sickle cell disease
A new distributional cost-effectiveness analysis of gene therapy versus standard-of-care for sickle cell disease (SCD) found that while gene therapy is cost-ineffective by conventional measures, it can be an equitable therapeutic strategy for persons living with SCD in the United States when equity, cost, and value of treatment are considered together. These findings highlight systemic health inequities faced by persons with sickle cell disease (SCD). The authors say this is the first quantitative consideration of health equity for patients with SCD regarding the decision between gene therapy and standard care and the first study of its kind in any rare disease.
UC San Diego Health Recognized for Health Equity in Care of Sickle Cell Crisis
UC San Diego Health has been awarded the prestigious 2022 California Association of Public Hospitals and Health Systems Quality Leaders Award in the category of health equity.
Even good gene edits can go bad
A Rice University lab is leading the effort to reveal potential threats to the efficacy and safety of therapies based on CRISPR-Cas9, the Nobel Prize-winning gene editing technique, even when it appears to be working as planned.
Research to Prevent Blindness and the American Academy of Ophthalmology Award New Research Grants to Improve Eye Care
Award recipients to use the American Academy of Ophthalmology IRIS® Registry (Intelligent Research in Sight) clinical database to improve care for all patients
CHOP Researchers Develop Proof-of-Concept Treatment that Elevates Both Adult and Fetal Hemoglobin
Researchers at Children’s Hospital of Philadelphia (CHOP) have developed a proof-of-concept treatment for blood disorders like sickle cell disease and beta-thalassemia that could raise hemoglobin levels by activating production of both fetal and adult hemoglobin. Using a viral vector engineered to reactivate fetal hemoglobin production, suppress mutant hemoglobin, and supply functional adult hemoglobin, the researchers developed an approach that could produce more hemoglobin through a single vector. The results were published in Haematologica.
Mount Sinai Ophthalmologists Develop New Technique to Assess Progression of Sickle Cell Retinopathy
(New York, NY – May 10, 2021)- Ophthalmologists at New York Eye and Ear Infirmary of Mount Sinai have created a new technique to evaluate patients with sickle cell retinopathy and assess the disease before it progresses and leads to…
Cecelia Calhoun Named Director of the Adolescent-Young Adult Sickle Cell Program at Yale Cancer Center and Smilow Cancer Hospital
Cecelia Calhoun, MD, MSPH, MBA, has been appointed Assistant Professor of Medicine (Hematology) and Assistant Professor of Pediatrics (Hematology/Oncology) at Yale Cancer Center and Smilow Cancer Hospital
Patients with Sickle Cell–Related Kidney Failure Benefit from Transplants But Are Less Likely to Receive Them
• Patients with kidney failure associated with sickle cell disease benefit from kidney transplants, but they’re less likely than other patients to receive them.
Gene therapy gives man with sickle cell disease the chance for a better future
In July 2019, Evie Junior enrolled in a clinical trial for an experimental stem cell gene therapy for sickle cell disease. The study is led by UCLA Broad Stem Cell Research Center physician-scientists Dr. Donald Kohn and Dr. Gary Schiller and funded by the California Institute for Regenerative Medicine.
Intellia Therapeutics Receives Grant to Develop Curative CRISPR/Cas9 In Vivo Sickle Cell Disease Treatments
Intellia Therapeutics Receives Grant to Develop Curative CRISPR/Cas9 In Vivo Sickle Cell Disease Treatments
Lost in Transition: Sickle Cell Clinic Helps Adults Continue Care
Networking California for Sickle Cell Care Initiative supports expansion of San Diego’s only adult sickle cell disease clinic at UC San Diego Health which offers child-to-adult transition health services to patients like Vanessa Hughes.
Understanding Sickle Cell Disease
Sickle cell disease is a lifelong inherited disorder of the red blood cells that poses many challenges for the people and families living with it. Rutgers Cancer Institute expert shares more on the basics of sickle cell disease and how the illness is managed.
Gut Microbiome Translates Stress Into Sickle Cell Crises
A new study shows how chronic psychological stress leads to painful vessel-clogging episodes—the most common complication of sickle-cell disease (SCD) and a frequent cause of hospitalizations. The findings, made in mice, show that the gut microbiome plays a key role in triggering those episodes and reveals possible ways to prevent them. The research was conducted by scientists at Albert Einstein College of Medicine and published online today in Immunity.
COVID-19: What you should know if you have sickle cell disease
If you have sickle cell disease, you are at higher risk than the general population for severe complications from COVID-19.
NIH announces $1 million prize competition to target global disease diagnostics
The National Institutes of Health has launched a $1 million Technology Accelerator Challenge (TAC) to spur the design and development of non-invasive, handheld, digital technologies to detect, diagnose and guide therapies for diseases with high global and public health impact. The Challenge is focused on sickle cell disease, malaria and anemia and is led by NIH’s National Institute of Biomedical Imaging and Bioengineering (NIBIB).
While Promoting Diseases Like Cancer, These Enzymes Also Cannibalize Each Other
In diseases like cancer, atherosclerosis, and sickle cell anemia, cathepsins promote their propagation. Drug trials to inhibit these enzymes have failed due to baffling side effects. Now a new study examines cathepsins in systems to remove some of the bafflement.
Yale Cancer Center researchers show identifying type of chronic pain in adults with sickle cell disease may lead to better outcomes
Identifying the type of pain an adult with sickle cell disease (SCD) experiences may be useful in improving treatment, according to a new study by researchers at Yale Cancer Center (YCC) and Smilow Cancer Hospital.
Likelihood of Prenatal Screening for Blood Disorders Varies Between Type of Healthcare Provider
A Rutgers Cancer Institute of New Jersey investigator and other collaborators examined prenatal screening practices for blood disorders between different types of care providers and found different variations in prenatal guidance.