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.

Type III Gaucher Disease is a lysosomal enzyme deficiency disorder caused by mutations in the GBA gene, resulting in impaired degradation of glucosylceramide and its accumulation in the liver, spleen, bone marrow, and central nervous system. Compared with Type I disease, Type III presents with progressive neurological involvement, including seizures, oculomotor abnormalities, and developmental delay. Without appropriate treatment, the disease continues to worsen, leading to significant functional decline and increased mortality risk.

Current treatment relies primarily on enzyme replacement therapy (ERT). However, ERT does not cross the blood–brain barrier (BBB) and therefore provides limited benefit for neurological manifestations. In addition, lifelong and frequent intravenous infusions impose a substantial burden on both patients and their families.

Sarcopenia is an age‑related progressive condition characterized by declining muscle mass, reduced strength, and functional deterioration. Loss of muscle mass increases the risk of falls, disability, hospitalization, and mortality, imposing long‑term health and quality‑of‑life burdens on middle‑aged and older individuals. Without treatment, sarcopenia progresses rapidly, affecting mobility, daily independence, and overall life expectancy.

Current management primarily depends on resistance training and nutritional supplementation; however, outcomes are frequently limited by adherence, individual variability, and chronic comorbidities. At present, no pharmacological therapy has demonstrated sustained and meaningful improvements in muscle mass and function.

Prader-Willi syndrome (PWS) is a rare genetic disorder caused by abnormalities in aspecific gene region on chromosome 15. It is characterized by early hypotonia andfeeding difficulties, followed by hyperphagia, rapid weight gain, short stature,reduced muscle mass, delayed sexual development, and cognitive or behavioralchallenges. Management is multidisciplinary, and growth hormone therapy is animportant treatment that supports growth and improves body composition.