How is Danon disease treated?

How is Danon disease treated?

Cardiac transplantation is a reasonable treatment for Danon disease despite the presence of muscular problems because the neurologic disability or problems are mild.

What are the symptoms of Danon disease?

Danon disease is a condition characterized by weakening of the heart muscle (cardiomyopathy ); weakening of the muscles used for movement, called skeletal muscles, (myopathy); and intellectual disability. Males with Danon disease usually develop the condition earlier than females and are more severely affected.

What diseases do vacuoles cause?

These diseases are a result of storage in cellular vacuoles and include Danon disease, Pompe disease, Fabry disease, and a form of HCM related to a mutation in the adenosine monophosphate (AMP)–activated, gamma-2 noncatalytic subunit of protein kinase (PRKG2).

How do you diagnose Danon disease?

Genetic testing of the LAMP2 gene is currently the gold standard for diagnosis and is available in specialized genetics laboratories. Most genetic mutations causing Danon disease predict reduced levels or even absence of the LAMP2 gene product, the LAMP-2 protein.

Is cardiomyopathy cardiovascular disease?

Overview. Cardiomyopathy (kahr-dee-o-my-OP-uh-thee) is a disease of the heart muscle that makes it harder for the heart to pump blood to the rest of the body. Cardiomyopathy can lead to heart failure. The main types of cardiomyopathy include dilated, hypertrophic and restrictive cardiomyopathy.

Is Danon disease fatal?

Danon Disease affects males more frequently than females. Untreated, the condition is fatal. Among males, the condition often results in a defective heart, weakening of muscles in the body, and mild to severe intellectual disabilities. A heart transplant is usually needed during childhood or early adolescence.

What happens when vacuole malfunction?

When a plant has been without water for a long time, the central vacuoles lose water, the cells lose shape, and the whole leaf wilts.

What will happen if vacuole is damaged?

If a cell did not have a vacuole, then it would be unable to carry out its usual functions and would eventually die. In plants, the vacuole plays an important role in water storage and the maintenance of structure.

Is there a cure for dilated cardiomyopathy?

Treatment of dilated cardiomyopathy depends on the causes. The goals of treatment are to reduce symptoms, improve blood flow and prevent further heart damage. Dilated cardiomyopathy treatment may include medications or surgery to implant a medical device that helps the heart beat or pump blood.

Who discovered Danon disease?

History. Danon disease was characterized by Moris Danon in 1981. Dr. Danon first described the disease in 2 boys with heart and skeletal muscle disease (muscle weakness), and intellectual disability.

What fluid is inside a vacuole?

cell sap
The fluid inside the vacuole is called cell sap. The fluid inside the vacuole is called cell sap.

Can a cell live without a vacuole?

What are the treatments for cardiomyopathy or arrhythmia?

Other procedures used to treat cardiomyopathy or arrhythmia include: Septal ablation. A small portion of the thickened heart muscle is destroyed by injecting alcohol through a long, thin tube (catheter) into the artery supplying blood to that area. This allows blood to flow through the area.

What are the treatment goals for cardiomyopathy?

Treatment for cardiomyopathy might not cure the problem with your heart. The main goals of treatment include: Your treatment may include medicines, procedures, and devices. You may need to see a cardiologist, a doctor who specializes in diagnosing and treating heart disease. The following medicines can help treat your symptoms.

How does an ICD work for cardiomyopathy?

This device monitors your heart rhythm and delivers electric shocks when needed to control abnormal heart rhythms. An ICD doesn’t treat cardiomyopathy, but watches for and controls abnormal rhythms, a serious complication of the condition. Ventricular assist device (VAD). This helps blood circulate through your heart.

What are the different types of cardiomyopathy surgery?

Types of surgery used to treat cardiomyopathy include: Septal myectomy. In this open-heart surgery, your surgeon removes part of the thickened heart muscle wall (septum) that separates the two bottom heart chambers (ventricles). Heart transplant.