Investigational drug restoreed parathyroid function in rare disease

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On Oct. 13, 2023, the National Institute of health (NIH) announced that an investigational drug, encaleret, restored calcium levels in people with autosomal dominant hypocalcemia type 1 (ADH1), a rare genetic disorder marked by an imbalance of calcium in the blood and urine, as well as abnormally low levels of parathyroid hormone, which regulates blood calcium levels. The results from the clinical trial were published in the New England Journal of Medicine.

In the mid-phase clinical trial, 13 participants with ADH1 received oral doses of the investigational drug for about 24 weeks. By the end of the trial, the treatment restored every participantメs blood calcium level to normal, and urine calcium approached normal levels. Levels of parathyroid hormone also normalized.

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