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Emcitate (tiratricol)

MCT8 deficiency

Last updated
78 sources

Product information and disease descriptionPlace of product in therapyApproaches to treatment

Heterogeneity of treatment effect

3 evidence topics · 3 sources

Treatment effect by functional impact of the SLC16A2 variant

“In these patients, Triac treatment according a predefined protocol with a median duration of 22.4 (IQR:12.9–42.4) months and a median dose of 40.5 (IQR:28.9–58.0) μg/kg was equally effective in reducing serum T3 concentrations, as well as improving clinical and biochemical outcomes irrespective of the functional impact of the underlying variant, leveraging the potential impact of treatment on thyrotoxic features to all patients” (opens the source at this quote in a new tab)

“We also showed equal effectiveness of the MCT8-independent T3 analogue Triac treatment on different thyrotoxic features across different LoF classifications.” (opens the source at this quote in a new tab)

Treatment targets by age

“Appropriate treatment targets may vary with age. For example, improved neurodevelopmental outcomes may be the most important goal in neonates but not adults; however, gaining body weight is beneficial to patients of all ages. Attaining these treatment targets may require different therapeutic strategies.” (opens the source at this quote in a new tab)

Phenotypic severity and residual transporter function

“It has been repeatedly reported that the severity of the clinical phenotype is related to the residual transport capacity of the mutated MCT8 protein.” (opens the source at this quote in a new tab)