Acoramidis

Acoramidis

Acoramidis is a pharmaceutical drug used in the treatment of transthyretin amyloidosis (ATTR), a rare and progressive disease caused by the misfolding of the transthyretin (TTR) protein. This misfolding leads to the accumulation of amyloid fibrils in various tissues and organs, causing a range of symptoms depending on the affected areas.

Mechanism of Action

Acoramidis works by stabilizing the TTR protein, preventing its misfolding and subsequent amyloid formation. By binding to the TTR protein, Acoramidis helps maintain its normal structure, thereby reducing the deposition of amyloid fibrils in tissues.

Indications

Acoramidis is indicated for the treatment of both hereditary and wild-type ATTR. Hereditary ATTR is caused by mutations in the TTR gene, while wild-type ATTR occurs without genetic mutations and is typically associated with aging.

Administration and Dosage

Acoramidis is administered orally. The dosage and treatment regimen depend on the severity of the disease and the patient's overall health. It is important for patients to follow their healthcare provider's instructions regarding the use of Acoramidis.

Side Effects

Common side effects of Acoramidis may include nausea, fatigue, and headache. Serious side effects are rare but can include liver toxicity and allergic reactions. Patients should report any unusual symptoms to their healthcare provider.

Clinical Trials

Acoramidis has undergone several clinical trials to evaluate its efficacy and safety in patients with ATTR. These trials have demonstrated that Acoramidis can significantly reduce the progression of the disease and improve the quality of life for patients.

See Also

• Transthyretin
• Amyloidosis
• Hereditary transthyretin amyloidosis
• Wild-type transthyretin amyloidosis

References

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