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Emcitate (tiratricol)

MCT8 deficiency

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78 sources

Product information and disease descriptionPlace of product in therapyApproaches to treatmentCurrent treatment options and standard of care

Symptom-directed neurological pharmacotherapy

3 evidence topics · 3 sources

Guideline recommendation for symptomatic neurological treatment

“We suggest that all patients should be offered empirical symptomatic treatment for neurological symptoms, including dystonia/hypertonia (spasmolytic drugs), drooling (e.g. anticholinergic drugs), and true epilepsy (carefully distinguished from a paroxysmal movement disorder); and should be referred to rehabilitation physicians/orthopedic surgeons/physiotherapists for measures anticipating contractures, scoliosis, a hip subluxation” (opens the source at this quote in a new tab)

“In the context of MCT8 deficiency, the effectiveness of such interventions has not been evaluated.” (opens the source at this quote in a new tab)

Symptomatic treatments by manifestation
ManifestationTreatmentConsiderations
Dystonia“Medications such as anticholinergics, L-DOPA, carbamazepine, or lioresal”“These therapies have shown no or mild efficacy.”
Drooling“Glycopyrolate or scopolamine”“Consider the ↑ risk for hyposalivation assoc w/↑ risk for dental caries”
Epilepsy“Standardized treatment w/ASM by experienced neurologist”“Many different ASMs may be effective; none has been demonstrated effective specifically for this disorder.”
Levodopa for dystonia

“Although dystonia is a prominent feature in MCT8 deficiency, current evidence does not support a dopaminergic mechanism underlying the motor symptoms. Consequently, levodopa is not considered a disease-modifying or standard treatment.” (opens the source at this quote in a new tab)