Allan-Herndon-Dudley syndrome is caused by mutations in the gene coding for a thyroid hormone transporter, preventing thyroid hormones from entering brain cells.
The Europe an Medicines Agency's Committee for Medicinal Products for Human Use has recommended granting marketing authorization for Emcitate for treating peripheralEmcitate was designated as an orphan medicinal product for treating AHDS in 2017. The Committee for Orphan Medicinal Products will now assess whether the orphan designations should remain.
Emcitate’s active ingredient, tiratricol, is an analogue of the thyroid hormone T3. It has a high affinity to thyroid receptors and does not require MCT8 to enter cells. It works similarly to T3 and can restore normal thyroid hormone activity in MCT8-dependent tissues. conducted in children and adults with AHDS in eight European countries and South Africa. Patients were treated with progressively increasing doses of tiratricol for up to 12 months.
Treatment-related adverse events occurred in 13% of patients and included excessive sweating, anxiety, nightmares, and irritability. Adverse reactions tended to occur at the beginning of treatment or when doses were increased, yet generally subsided during treatment.
Thyroid Disease Thyroid Dysfunction Hyperthyroidism Thyroid Thyrotoxicosis Genomics Genomic Medicine Brain Europe European Genetic Disorder Arrhythmia Heart Failure Heart Failure (HF) Genetics Grant Heart Anxiety Anxiety Disorder Blood Children Child Childhood Pediatrics Kids Disability Hospitals Muscles Nightmares Receptors Sepsis Bloodstream Infections
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