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Penn Vet Researchers Identify Effective Treatment

Penn Vet Researchers Identify Effective Treatment for Niemann Pick Type C
UPenn - 2/25/15

Niemann Pick Disease type C, or NPC, is a disease most people have never heard of, affecting just one person in 150,000. Yet the disease is a devastating one. Frequently diagnosed in children in their elementary school years, sufferers usually die by the time they’re 20. The disease is sometimes referred to as “childhood Alzheimer’s” because of the progressive mental and physical decline seen in the children it afflicts.

There is no specific, FDA-approved treatment for NPC, only drugs to treat the symptoms. That is now changing.

A study coming out in Science Translational Medicine and led by University of Pennsylvania School of Veterinary Medicine researchers has shown that cats with NPC — which mirrors the human version of the disease — show vast improvements when treated with a compound called cyclodextrin. While NPC typically results in inexorable neurological decline, administering cyclodextrin into the fluid around the cats’ brains largely halted the progression of disease.

This study showed that the treatment was safe enough to begin a clinical trial in children, which launched last year.

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Support Of Accelerated Research for Niemann-Pick C, or SOAR-NPC, was created by parents and scientists as a grassroots response to the absence of treatments for this terminal disease.

The number one goal of SOAR-NPC is to work as quickly as possible to find evidence-based treatments and ultimately a cure for Niemann-Pick Type C disease, a rare neurological illness.

SOAR scientists are internationally recognized in NPC research from institutions including Washington University in St. Louis, Albert Einstein College of Medicine, and University of Pennsylvania.  SOAR family foundations are dedicated to raising money and awareness to combat this deadly disease.

 
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