What is Niemann-Pick Type C?

Niemann-Pick Disease Type C (NP-C) is a devastating neurodegenerative disease caused by an accumulation of lipids (fats) in the liver, brain and spleen. In this video Professor Marc Patterson (Professor of Neurology, Pediatrics, and Medical Genetics, Mayo Clinic) and Professor Paul Gissen (Honorary Consultant in Paediatric Metabolic Diseases, Great Ormond Street Hospital) explore the causes, symptoms, and potential therapies for Niemann-Pick disease type C.
At NPUK we are a charitable organisation dedicated to making a positive difference to the lives of those affected by Niemann-Pick diseases. Our central goals are to raise much needed awareness, provide practical and emotional support, advice and information and facilitate research into potential therapies for NPD.
To learn more about Niemann-Pick UK (NPUK) and Niemann-Pick disease, please visit us at our site: http//www.npuk.org

[@toystorylover5257]

Thank you for the information! It is greatly appreciated!

[@hayatahmadroomi9263]

Thank you so much, sir.

[@danidevic]

Very good information!

[@ishakkocatepe7658]

hey ihave the npc1 type c pleasease
cut your please give me a list of the possiebel consuquentes of having the npc1

[@YESTOYOURWILL]

Thank you for this my grand daughter was recently diagnosed with this

[@mahsayavari176]

Hi
What was the treatment?

[@YESTOYOURWILL]

Currently she has had no treatment. We just received miglustat and will be starting this to help slow down her progression. We do a lot of praying on her behalf and all the kids dealing with these diseases and disorders. Jesus has really been our source of healing this far. I pray no one else will have to go through what these kids have been through. May God bless you and have mercy on us all

[@williamspaul8064]

Meeting Dr Dagba on UA-cam has really been one of the best day of my life after years of suffering I have been finally cured from Heart Disease , Thanks doc God bless you.

[@chanabayla1823]

Most cases hit mostly Jewish ashkenazi. We automatically get tested for most of the "Jewish" dieases. Doesn't matter if you are Jewish or not u have a responsibility to me as a parent to get tested as most of these dieases are fatal