Niemann-Pick Disease Type C Treatment; a Medication Called Miglustat (Zavesca) May Be an Option

 

Niemann Pick Disease Type C

No effective Niemann-Pick disease type C treatment is available for people with type A or B, but for people with mild to moderate type C, a medication called miglustat (Zavesca) may be an option. However, there is no cure for Niemann-Pick disease. Children usually die from infection or progressive neurological loss. Niemann-Pick is a rare, inherited disease that affects the body's ability to metabolize fat (lipids and cholesterol) within cells. Niemann-Pick disease can affect the bone marrow, spleen, liver, nerves, brain, and, in severe cases, lungs. Niemann-Pick type C is a lysosomal storage disease associated with mutations in NPC1 and NPC2 genes.

Niemann-Pick type C (NPC) is a slowly progressive disorder whose principal manifestations are age dependent. In infants, Niemann-Pick disease type C leads to various disorders such as Gaucher disease, idiopathic neonatal cholestasis, alpha-1-antitrypsin deficiency, tyrosinemia, and various congenital infections. According to the National Organization for Rare Disorders 2020 report, the frequency of NPC is similar in both men and women, wherein it is estimated to occur in 1 in 100,000-120,000 live births. It is also known as Childhood Alzheimer's, and as with Alzheimer's disease, there is no current Niemann-Pick disease type C treatment available.

However, there are some promising new treatments being tested in clinical trials for the symptoms of this condition. For instance, in February 2020, Cyclo Therapeutics announced the completion of enrollment in its phase 1/2 trial to evaluate the tolerability, safety, and efficacy of Trappsol Cyclo for Niemann pick disease type C treatment. Niemann-Pick disease is divided into four main types, such as type A, B, C1, and C2. These types are classified on the basis of genetic cause and the signs and symptoms of the condition. Type C1 is the most common form of the disease and type C2 is a rare form of the disease. However, there is currently no cure for Niemann-Pick disease.


Location: India

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