An oral therapy to treat Wolfram syndrome significantly improved pancreatic functions and induced improvement in symptoms at 24 and 48 weeks for most participants in a phase 2 trial.
There are no FDA-approved treatments for Wolfram syndrome, a rare neurogenerative disease that impacts multiple organ systems, according to Fumihiko Urano, MD, PhD, the Samuel E. Schechter Professor of Medicine and professor of pathology and immunology at Washington University School of Medicine. In findings from the HELIOS trial presented at the Joint Congress of the European Society for Paediatric Endocrinology