TRND (Therapies for Rare and Neglected Diseases) and the NIH team met with FDA representatives on December 13^th to discuss the progressive plans to bring Cyclodextrin to a clinical trial in 2012. For TRND, Niemann-Pick Type C is one of six pilot projects that were selected.

The NNPDF does a wonderful job of keeping families and people interested up to date on how things are progressing in the disease community for Niemann-Pick Type C. You can visit updates on their page here: http://www.nnpdf.org/aboutus_14.html Continue Reading

You possibly can recall someone you knew that suffered from Alzheimer’s disease. Currently 5.3 million Americans are living with this progressive and fatal brain disease with no cure (www.Alz.org). Alzheimer’s is progressively devastating in how it destroys brain cells, causes loss of memory (progressive dementia), and problems with intellectual functions that affect many of the things you and I take for granted. Alzheimer’s is the seventh leading cause of death in the United States (www.Alz.org).

In comparison to Niemann-Pick Type C Disease (NPC), it has maybe less than a 100 people currently living in the U nited States. Many sources state that 500 cases have been diagnosed ever worldwide. That’s not exactly as common of disease as Alzheimer’s.  NPC is described as a genetic degenerative neurological  disorder that mostly strikes during childhood that stores fatty lipids in the brain, liver, spleen, and bone marrow because of the cells inability to metabolize cholesterol. Although uncommon, adult onset can take place with NPC. Outcome is also fatal with no cure. Continue Reading

How many times have you been looking for something at your house but you accidentally find something you previously were missing? Wouldn’t it be a shame if the prize you were seeking was within reach but you discounted that it could be that easy? What if understanding Niemann-Pick Type C disease opened up the door to help millions of Americans with other disorders involving cholesterol? Of course nothing in life is easy nor will it always be within reach. With being human comes the tendency to make oversights. Continue Reading