Name: Emcitate (tiratricol) in MCT8 deficiency: Long-term morbidity
Published: 2026-09-14T15:39:06-04:00

Product information and disease description Place of product in therapy Disease description

Long-term morbidity

5 evidence topics · 2 sources

Progressive motor complications

“Progressive hypertonicity of the limbs with brisk reflexes, ankle clonus, and extensor plantar responses (Babinski sign) leads to spastic quadriplegia and joint contractures. Overall muscle mass (particularly proximally) is reduced and associated with generalized muscle weakness.” (opens the source at this quote in a new tab)
“Pectus excavatum and kyphoscoliosis are most likely the result of hypotonia and reduced muscle mass.” (opens the source at this quote in a new tab)

Cardiovascular morbidity and sudden death

“Sudden death is one of the leading causes of mortality in individuals with MCT8 deficiency and may be linked to cardiac events. In a large retrospective cohort study, a wide spectrum of cardiac abnormalities was observed in these individuals, including premature atrial contractions, elevated blood pressure, and resting tachycardia.” (opens the source at this quote in a new tab)

Feeding difficulties, undernutrition, and respiratory infection

“Feeding difficulties are among the most frequent and functionally limiting complications in patients with MCT8 deficiency.” (opens the source at this quote in a new tab)
“Low body weight is frequently observed in individuals with MCT8 deficiency and likely reflects the combined effects of increased basal metabolic rate and dysphagia secondary to axial hypotonia and progressive muscle wasting.” (opens the source at this quote in a new tab)
“Furthermore, respiratory infections, a leading cause of mortality in this population, are more frequent in undernourished patients, suggesting a link between impaired nutritional status and increased susceptibility to infection” (opens the source at this quote in a new tab)

Bone health and fracture risk

“Elevated TH levels in adulthood have been associated with accelerated bone turnover, reduced bone mineral density, increased fracture risk, and osteoporosis. Individuals with MCT8 deficiency are typically immobile, which may further contribute to their elevated risk of fractures” (opens the source at this quote in a new tab)

Liver function abnormalities

“Some individuals with MCT8 deficiency exhibit abnormal liver function markers, such as elevated SHBG or transaminase levels.” (opens the source at this quote in a new tab)