Correct Answer : Both 1 and 2
* Children suffering from lysosomal storage disorders like Gaucher disease are facing a bleak future as their treatment has been stopped due to the exhaustion of one-time support from the Union health ministry.
* It is an inherited lysosomal storage disorder (LSD).
* It is a type of disease that causes fatty substances (sphingolipids) to build up in your bone marrow, liver and spleen.
* The sphingolipids weaken bones and enlarge your organs.
Symptoms : Enlarged spleen, liver, eye movement disorders and yellow spots in the eyes etc.
There are three types of Gaucher disease :
Type1 : It affects your spleen, liver, blood and bones. It doesn’t affect your brain or spinal cord. Gaucher disease type 1 is treatable, but there’s no cure.
Type 2 : A rare form of the disorder appears in babies younger than 6 months old.
* It causes an enlarged spleen, movement problems and severe brain damage. There’s no treatment for Gaucher disease type 2.
Type 3 : It is the most common form which appears before age 10 and causes bone and organ abnormalities and neurological (brain) problems.
* Treatments can help many people with Gaucher disease type 3 live into their 20s or 30s.
Treatment : There’s no cure for Gaucher disease, but treatments can relieve symptoms and greatly improve quality of life.
