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
Hong Kong-based Insilico Medicine published research Monday showing that its deep learning system could identify potential treatments for fibrosis. That system, called generative tensorial reinforcement learning, or GENTRL for short, was able to find six promising treatments in just 21 days, one of which showed promising results in an experiment involving mice. The research has been
Four hundred and eight early-career researchers have been awarded European Research Council grants in this year’s first completed ERC call for proposals. The highly-coveted funding will help individual scientists and scholars to build their own teams and conduct pioneering research across all disciplines. The grants, worth in total €621 million, are part of the EU’
Chronicling the EU’s past struggles and new efforts in promoting gene therapies, known in Europe as advanced therapy medicinal products In Europe, legislation relating to medicines based on genes, tissues, or cells dates back nearly 20 years, and was reinforced by specific European Union regulation that came fully into effect nearly a decade ago—but still today
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,
NHS Digital said that a recently undertaken project has shown how digital health tools helped in enhancing access to healthcare for patients suffering from hearing and sight loss. The project taken up under NHS Digital’s Widening Digital Participation (WDP) Programme enabled people with sensory impairments in West Yorkshire and Harrogate gain awareness on using digital
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
The next generation of waterproof smart fabrics will be laser printed and made in minutes. That’s the future imagined by the researchers behind new e-textile technology. Scientists from RMIT have developed a cost-efficient and scaleable method for rapidly fabricating textiles that are embedded with energy storage devices. In just three minutes, the method can produce
New research reveals a new role for the enzyme telomerase, which scientists thought was turned off in most normal adult cells, except in cancerous tumors where it promotes unlimited cell division. The researchers discovered that as normal, healthy adult cells approach cell-death, they produce a burst of telomerase that prevents malignancies and softens the final
Researchers identify nearly 8,000 strains of bacteria, while also clarifying their genetic and metabolic context. The human digestive tract is home to thousands of different strains of bacteria. Many of these are beneficial, while others contribute to health problems such as inflammatory bowel disease. Researchers from MIT and the Broad Institute have now isolated and
