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What Is ‘Childhood Alzheimer’s’? New Treatment Offers Hope To Teens With Rare Disease

A rare disease known as Niemann-Pick disease type C (NPC), which afflicts one in 150,000 children and teenagers, is largely unheard of but leaves patients and families in a state of desperation. If diagnosed, a child will experience quick and progressive mental and physical deterioration over the course of several years - similar to Alzheimer's disease, hence its nickname of "childhood Alzheimer's."

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Dr. Sears Comments:

Dr. Barry Sears
Neimann-Pick disease is caused by the accumulation of sphingolipids in the brain.  A number of other neurological diseases are caused by other sphingolipids.  It is unfortunate, but I find it highly doubtful this drug will make a difference because these are genetic diseases that disrupt the normal metabolism of sphingolipids and the drug has little likelihood of crossing the brain-brain-barrier in the first place.

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