Sotagliflozin is a drug being developed to lower blood sugar levels in type 1 diabetes by increasing the amount of sugars excreted in the urine. It is taken once a day tablet in conjunction with insulin to prevent large rises and falls in blood sugar levels. This may be especially useful for people who cannot control their blood sugar levels with insulin alone. Sotagliflozin is currently in clinical trials which have shown that sotagliflozin has the potential to reduce and control blood sugar levels when taken with insulin.
Eladocagene exuparvovec is a type of gene replacement therapy which involves the transfer of the gene encoding the production of the enzyme needed by the brain for the formation of dopamine and serotonin. The gene therapy is injected via a surgical procedure into an area of the brain called the putamen. By increasing production of the AADC-enzyme, this therapy increases dopamine production in the target area of the brain and improves motor and cognitive symptoms in patients. If licensed, eladocagene exuparvovec will provide the first medicinal treatment option for adult and child patients with AADC-deficiency, a disease of very high unmet clinical need.