The FDA unveils a “plausible mechanism pathway” to speed approval of innovative therapies for ultra-rare diseases, paving the way for personalized and gene-based treatments.

Japanese researchers restored movement in a paralyzed man using reprogrammed stem cells injected into his spinal-cord injury. The treatment, tested in four patients, helped one man stand independently and another regain limb movement, offering early evidence that iPS cell therapies may support neural repair and future paralysis treatments.

Stanford Medicine researchers cured Type 1 diabetes in mice using a gentle immune-reset approach that combines donor blood stem cells and pancreatic islet transplants. The method prevented autoimmunity, required no immunosuppressive drugs, and may pave the way for future treatments for diabetes and other autoimmune diseases.