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14 results for "sickle cell disease"
Nemours Children's Hospital in Wilmington can now offer a groundbreaking one-time gene therapy that could give children with sickle cell disease a chance at a transformed life. The FDA-approved treatment addresses the root genetic cause of the disease that affects 100,000 Americans and shortens life expectancy by more than 20 years.
Uganda is raising $4 million to build its first bone marrow transplant center, offering hope to 20,000 children born annually with sickle cell disease. The facility could cure patients who currently face $300,000-500,000 treatment costs abroad.
Children with sickle cell disease now have a survival rate exceeding 95% thanks to breakthrough stem cell transplant techniques. New treatments are also opening curative options for kids who previously had no donor matches.
Austin Louis spent his entire life in excruciating pain from sickle cell disease until a revolutionary gene therapy treatment made him feel like "a new human." The 20-year-old New Jersey man became the first patient at Children's Hospital of Philadelphia to receive the FDA-approved CRISPR treatment outside clinical trials.
A Ugandan biotech firm just received a U.S. patent for a gene therapy that could treat sickle cell disease for 95% less than current cures. The breakthrough could finally bring life-saving treatment to 20 million patients worldwide who can't afford existing options.
After decades of research, gene therapy is bringing lasting relief to sickle cell patients after just one treatment. UCLA researchers are leading the charge to cure a disease that affects 100,000 Americans.
After becoming the first country to approve a revolutionary gene-editing therapy in 2023, the UK is now using Casgevy to treat patients with sickle cell disease and β-thalassaemia. The treatment has shown remarkable results, freeing 97% of sickle cell patients from painful episodes for over a year.
Victoria Hospital in Kisumu has become Kenya's first public facility to offer automated red blood cell exchange therapy for sickle cell patients. The specialized machine offers hope to thousands in western Kenya, where the disease is highly prevalent.
Serenity Cole, 18, spent years in constant pain from sickle cell disease. Now she's pain-free thanks to a revolutionary payment deal where drug companies only get paid if their $2-3 million gene therapies actually work.
Serenity Cole, 18, spent her recent Christmas making crafts with family instead of lying in a hospital bed. A groundbreaking Medicaid payment model made her potentially life-changing gene therapy possible.
Medicaid programs across America are using a revolutionary payment model for sickle cell gene therapy: drug companies only get full payment if the treatment actually works. Serenity Cole, 18, is living proof it does, celebrating her first pain-free Christmas in years after becoming one of the first patients treated under the new system.
Eighteen-year-old Serenity Cole spent Christmas with family instead of in the hospital, thanks to a groundbreaking gene therapy that could cure her sickle cell disease. The federal government is now making states pay for the $2-3 million treatment only if it actually works.
A foundation leader living with sickle cell disease is championing a plan to teach students about the genetic condition before they start families. The comic strip education model has already shown success in pilot programs across Ghana.
Scotland just approved a groundbreaking gene therapy that lets sickle cell patients get treatment without needing a matching donor. Four other life-changing treatments for cancer and blood disorders also got the green light.