New Treatments May Be Available for Niemann-Pick Disease Soon

Joslyn Crowe, Executive Director of the National Niemann-Pick Disease Foundation (NNPDF), talks about the programs the foundation has developed to help families be prepared when treatments get approved for Niemann-Pick disease.
Niemann Pick disease is a series of rare lysosomal storage disorders in which toxic quantities of lipids accumulate in the spleen, liver, lungs, bone marrow, and brain. Niemann-Pick disease is divided into four main types: type A, type B, type C1, and type C2. These types are classified on the basis of genetic cause and the signs and symptoms of the condition.
Type A and Type are caused by mutations in the SMPD1 gene while types C1 and and C2 are due to mutations of the NPC1 and NPC2 genes, respectively.
The U.S. Food and Drug Administration is currently reviewing several promising therapeutics to treat Niemann-Pick disease type C.
For more information about this and other lysosomal disorders, goto checkrare.com/lysosomal-storage-disorders/.

[@ArmanKhan-li7co]

kindly update if you ppl found any treatment for niemen pick disease .. my daughter is effected and it will be a great that i can start treatment for her ...

[@gajendrab8383]

My son also have type c and undergone BMT

[@juanpedrolopezmartinez5380]

i need the treathment i am 34 years old and i losing my personality and capacity to talk. Please.. help us

[@Payitforward-xt1kr]

Hopefully this company can help Juan. God Bless brother

[@r.i.tutorial8264]

Is treatment now available

[@captainrahil2446]

When treatment will come for Neiman pick dieses A/B ?
I am from India

[@ArshadAli-db3yx]

My baby is of three years old and is suffered with neimanpick disease type c can you any help to me?

[@gajendrab8383]

You Can meet dr Sunil Bhat in Narayana Hrudayalaya Bangalore