Increased Time to Hypoglycemia and Reduction in Cornstarch Use in Cohorts 1 and 2; Improvement of Additional Key Metabolic Measures Observed Cohort 1 Patients Continue to Demonstrate Long-Term, Durable Responses Company to Enroll Three Patients in Expansion Cohort to Confirm 6.0 × 10^12 GC/kg Dose as Optimal Dose for Phase 3 Study Ultragenyx Pharmaceutical, a
Breaking down how the gene editing technology is being used, for the first time in the United States, to treat patients with severe medical conditions Source Smithsonian.com Last fall, the birth of genetically edited twin girls in China—the world’s first “designer babies”—prompted an immediate outcry in the medical science community. The change to the twins’ genomes,
It was a crucial moment when NHS England and the National Institute for Health and Care Excellence (NICE) approved Chimeric Antigen Receptor T-cell (CAR-T) therapies for use on the NHS last year. CAR-T therapy uses a patient’s own white blood cells to fight cancer – England became one of the first countries in the world to make
Early clinical trials of a gene therapy for aromatic amino acid decarboxylase (AADC) deficiency have yielded encouraging results for its safety and efficacy, a study highlights. But researchers call attention to the importance of a timely diagnosis for treatment success, as only early interventions could fully prevent long-term brain damage and other outcomes. Their study, “Aromatic amino
Clusters of living brain cells are teaching scientists about diseases like autism. With a new finding, some experts wonder if these organoids may become too much like the real thing. Source The New York Times Two hundred and fifty miles over Alysson Muotri’s head, a thousand tiny spheres of brain cells were sailing through space. The clusters,
New research published in the ‘Journal of Dental Research’ has shed light on the science behind the formation of the periodontal ligament Our teeth take thousands of bites per day, and understanding exactly what holds them in place and how is key to helping people live with their own teeth for longer. Now new research
Harvard scientists have combined organ-on-a-chip and stem-cell technologies to make a powerful tool for diabetes research and beta-cell transplantation By combining two powerful technologies, scientists are taking diabetes research to a whole new level. In a study led by Harvard University’s Kevin Kit Parker and published in the journal Lab on a Chip on Aug. 29, microfluidics and human,
A promising new treatment for cancer patients has received approval from the Centers for Medicare and Medicaid Services, paving the way for more patients to get the treatment known as CAR T-cell therapy. While this decision will likely expand access, particularly for some lymphoma and leukemia patients, treatment remains prohibitively expensive, prompting some patients to opt for experimental rather
A research team from Osaka University said Thursday it had conducted the world’s first transplant of corneal tissues using artificially derived stem cells in July. The university’s team, led by Koji Nishida, may have created a new treatment for those suffering from corneal disease, as current treatment procedures involve waiting for corneal donations from deceased
The use of human embryonic stem cell (hESC)-derived Retinal Pigment Epithelium (RPE) transplants has advanced dramatically in different forms for clinical application in macular degeneration. This review focuses on the first generation of hESC-RPE cell line, named as “MA09-hRPE” by Astellas Institute of Regenerative Medicine (AIRM), and its therapeutic application in human, which evaluated the
