All articles tagged: Sickle-cell disease without crisis (D57.1)

Gene therapy for sickle cell disease (SCD) below $2 million is likely to be cost-effective, according to a study published online Jan. 23 in the Annals of Internal Medicine.Anirban Basu, Ph.D., from The Comparative Health Outcomes, Policy & Economics Institute at the University of Washington in Seattle, and colleagues examined the c

COVID-19 immunization completion is nearly half for people with versus without sickle cell disease (SCD), according to a research letter published online Jan. 8 in JAMA Network Open.Hannah K. Peng, from the University of Michigan in Ann Arbor, and colleagues assessed COVID-19 immunization coverage among 3,424 Michigan residents wit

After approving Casgevy (exagamglogene autotemcel) in December to treat sickle cell disease, the U.S. Food and Drug Administration announced Tuesday that the therapy has now been approved to treat patients older than 12 years with transfusion-dependent beta-thalassemia.Casgevy is the first CRIS

The annual meeting of the American Society of Hematology was held from Dec. 9 to 12 in San Diego and attracted participants from around the world, including hematology specialists as well as clinical practitioners and other health care professionals. The conference featured presentations focusing on the diagnosis, treatment, and

The FDA has approved the first-ever treatment for sickle cell disease using gene editing. This groundbreaking development marks a significant milestone in medical science. The treatment involves altering patients' DNA to address the underlying genetic cause of the disease, offering hope for a cure and improved quality of life for those suffering from this debilitating condition.

Two milestone gene therapies for sickle cell disease have been approved by the U.S. Food and Drug Administration.Casgevy is the first medicine available in the United States to treat a genetic disease using the CRISPR gene-editing technique. The one-time treatment permanently changes DNA in a patient's blood cells, freeing them from the excruciat

From 2014 to 2021, uptake of disease-modifying treatments (DMTs) for sickle cell disease (SCD) remained low, according to a study published online Nov. 22 in JAMA Network Open.Terri Victoria Newman, Pharm.D., from the University of Pittsburgh, and colleagues examined characteristics that may be associated with DMT use among patients wit

For pediatric patients with sickle cell disease (SCD), ophthalmologic complications include nonproliferative retinopathy (NPR) and proliferative retinopathy (PR), which occur in 33 and 6 percent, respectively, according to a study presented at the annual meeting of the American Academy of Ophthalmology, held from Nov. 3 to 6 in San Francisco.Ba

The FDA is evaluating a groundbreaking sickle cell disease treatment using CRISPR technology, potentially the first of its kind to be approved. The treatment, known as exa-cel, could eliminate the need for donor bone marrow transplants by editing patients' DNA to correct the mutation causing the disease. With the FDA's final decision expected by December 8, the treatment's efficacy and safety rema

A new gene therapy for sickle cell disease was deemed safe by a U.S. Food and Drug Administration advisory panel on Tuesday, paving the way for full approval by early December.The FDA had already decided that the therapy, known as exagamglogene autotemcel (exa-cel), was effective. Developed by Vertex Pharmaceuticals of Boston and CRISPR Therap

Patients with sickle cell disease may soon have two new treatments to try. On Tuesday, a U.S. Food and Drug Administration advisory committee will weigh the merits of a new gene therapy for the painful, inherited condition. The agency is expected to make a decision on that therapy in early December, and it also plans to decide on a second new treatment