Niemann Pick Disease is a type of genetic medical condition. The disease mainly occurs in four different variants i.e. types A, B, C, and D. Niemann Pick disease leads to different medical problems, while it advances rapidly. The symptoms and adverse effects mainly take place in each of the variant of the disease often take place because of the formation of sphingomyelin i.e. a specific type of body fat. Unfortunately, Niemann Pick disease does not have any definite cure and the patients often experience substantial death and illness even at their young ages. (1)
What Is the Best Treatment For Niemann Pick Disease?
Until now, doctors are unable to find any specific treatment to cure the problem of Niemann Pick Disease. However, in some cases, depending on the specific type of the Niemann Pick disease, treatment varies largely, according to which-
Type A of Niemann Pick Disease
Doctors do not know about any treatment to cure the problem of type A Niemann Pick disease at this moment. However, one can manage the symptoms with supportive care.
Type B of Niemann Pick Disease
Doctors have different treatment options to cure type B Niemann Pick disease. These include enzyme replacement therapy, bone marrow transplantation, and gene therapy. Along with this, doctors are doing consistent and ongoing research work to determine the effectiveness related to each of these treatments.
Type C of Niemann Pick Disease
Patients with type C Niemann Pick disease may get relief by physical therapy treatment, as it helps in easy mobility. Also, doctors use Miglustat medication i.e. an enzyme inhibitor to treat the problem of type C of Niemann disease. The enzyme inhibitor mainly avoids the patient’s body from producing a fatty substance called sphingomyelin in the body. Here, the fatty substance is obtained mainly from cholesterol. (4)
Key Facts of Niemann Pick Disease
- Niemann Pick disease is a type of genetic disease and it results in the accumulation of fatty products within the cells and it eventually causes death.
- Symptoms of the disease have close relations with various types of fatty substances, which accumulate, the specific affected organs and to the extent of the damage.
- A few of the children suffering from Niemann Pick disease fail to survive during their early childhood, while others with specific forms may easily survive up to adulthood.
- Children often die because of the progressive disease or infection of the nervous system.
- Until now, doctors have not found any specific treatment to cure the disease. (2)
Types, Causes, and Occurrence of Niemann Pick Disease
Niemann Pick Disease of type A and type B take place whenever body cells do not have the ASM i.e. acid sphingomyelinase enzyme. This substance metabolizes or breaks down the fatty substance of sphingomyelin, which remains present in almost everybody cell. If your body cells do not have ASM or it fails to function properly, the formation of sphingomyelin takes place within the cells. This destroys the body cells and creates difficulty in the regular function of your body organs.
- Type A and Type B Niemann Pick Disease take place in almost every type of race and ethnicity. However, it is common among Ashkenazi people.
- Type C Niemann Pick Disease takes place whenever the body fails to break down lipids/fats and cholesterol properly. This results in excessive cholesterol in the spleen and liver, which excessive accumulation of various other lipids in the human brain. Type C Niemann Pick Disease is common among people of Spain and Puerto Ricans.
- Type C1 is a type C variant and it involves a defect interfering with the cholesterol movement between different brain cells. Type C1 Niemann Pick Disease is common among Canadian and French people. (3)
To conclude, we should say that Niemann Pick Disease is a type of genetic disease and it takes place mainly in different types. These type A NPD, type B NPD, type C NPD, and type D Niemann Pick Disease. Because of this, the treatment to cure Niemann Pick Disease depends solely on the specific type, even though until now, doctors have failed to come up with the best NPD treatment.
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