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Pfaffle, R

The wonders of 2-deoxy-D-glucose

Kidney transplantation and cystine-depleting therapy with oral and topical cysteamine have transformed cystinosis from a universally fatal childhood disorder into a treatable chronic disease, with many adults achieving normal lives

A number of other approaches for treating GA have failed in human trials (e.g., visual cycle inhibitors, emixustat, Acucela, sustained release of neurotrophic factors, NT501, Neurotech, and complement pathway inhibitors, eculizumab, Soliris) 7,9

13 males) in the ID group and 30 participants (mean age = 34.67 13.68 years

This positive feedback loop is interrupted by T4 monotherapy since it over-suppresses the TSH and thereby over-reduces T4-to-T3 conversion in the thyroid gland and throughout the body