Wilson’s Disease is a rare inherited metabolic disorder caused by ATP7B gene mutations that impair normal copper excretion, resulting in progressive accumulation in the liver, brain, and other organs. The rising copper burden contributes to hepatitis, cirrhosis, neurological deterioration, and psychiatric symptoms. Without timely intervention, the disease continues to worsen and may lead to fatal liver failure or irreversible neurological impairment.

Current treatment primarily relies on chelating agents to remove excess copper. However, existing therapies are often limited by dose‑dependent adverse effects, gastrointestinal intolerance, and frequent dosing, all of which reduce long‑term adherence. Some patients also exhibit inadequate response in later stages of disease and cannot further escalate dosing.

Cardiomyopathy is a heterogeneous group, which defined by American Heart Association (AHA), of myocardial diseases characterized by structural and functional abnormalities of the heart muscle. It is defined as an anatomic and pathologic diagnosis involving mechanical or electrical dysfunction, typically resulting in inappropriate ventricular hypertrophy or dilatation. These conditions often progress to chronic heart failure, leading to significant morbidity and mortality.

Current treatment approaches—including beta‑blockers, calcium channel blockers, and newer myosin inhibitors—aim to reduce myocardial oxygen demand and alleviate symptoms. However, many patients continue to experience residual symptoms, dose‑limiting side effects, and ongoing disease progression.