4 results
1. Normalization of the abnormal serum TH parameters and thereby improving the clinical condition of the AHDS patients2. Observation of changes in cognitive and motor function.
Primary Objective:• To evaluate the effects of withdrawal of tiratricol treatment (placebo group) on serum total triiodothyronine (T3) concentrations, measured by liquid chromatography with tandem mass spectrometry (LC/MS/MS), and the requirement…
Part I:Primary Objective: Evaluate the effects of tiratricol on neurodevelopment in young MCT8 deficiency patients, as measured by the Gross Motor Function Measure (GMFM)-88 and Bayley Scales of Infant Development (BSID-III) Gross Motor Skill Domain…
Objectives (all are exploratory):• To measure and analyze 89Zr-Df-IAB22M2C uptake cluster of (CD8+ PET signal) at and between baseline and post-treatment in tumor lesions and reference normal tissues, including T-cell rich tissues, using PET imaging…