Treatment of Wolfram Syndrome Type 2 With the Chelator Deferiprone and Incretin Based Therapy
Status:
Unknown status
Trial end date:
2018-12-01
Target enrollment:
Participant gender:
Summary
Patients who are genetically diagnosed with the recently reported and rare Wolfram syndrome
type 2 ( WFS2) and have the degenerative and symptomatic disease including signs such as
diabetes, platelet aggregation defect or visual problems will be asked to participate in this
study. Knowing the pathomechanism of WFS2 with rapid cell death, after doing baseline
investigations to asses the severity of their disease, the participants will be offered a
chelator therapy with in addition to the antioxidant Acetylcystein, in diabetic patients an
Incertin (GLP-1 ) therapy will be offered as well. The baseline investigations will be
repeated after 2 months and after 5 months of therapy in order to asses the progression of
the disease and to show if the chelator and anti oxidant therapy and in diabetic patients the
GLP-1 therapy could stop the progression of the disease.