Canada has a lot to celebrate for their current and future patients living with the “Childhood Alzheimer’s” or Niemann-Pick Type C (NPC). During the last full week of March 2010, the announcement came down that Health Canada [The Food Directorate, the Therapeutic Products Directorate and the Natural Health Products Directorate in the Health Products and Food Branch (HPFB)] approved the official use of Zavesca (Miglustat). Before now the only approved use was for Gaucher’s Disease Type 1. Continue Reading

Dementia, who was the thief that thought up this bright idea? Sorry honey, what did you say again? OK that might be a case of selective hearing. We’ve all have had family or known people who have suffered with this horrible symptom. When you mention dementia, Alzheimer’s comes to mind or we all tend to think of an elderly person with their hands on their head not able to remember out of frustration pounding the table with their fists. STOP!… WAIT!… Did you know our children can suffer from dementia too? 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

On March 9, 2010 the FDA has requested additional preclinical and clinical information regarding Zavesca (Miglustat) before giving the ultimate approval that the Niemann-Pick Type C community was hoping for. Actelion stated that they will work diligently with the FDA to explore the best ways to address the points raised by the agency.

Jean-Paul Clozel, M.D. and Chief Executive Officer of Actelion commented: “We remain committed to bringing an approved treatment to patients suffering from NP-C disease and in this spirit we will continue the dialogue with the FDA.” Continue Reading

Social Security adds 38 new medical conditions that qualify for disability help.  The new conditions range from early-onset Alzheimer’s disease to rare diseases that primarily affect children which includes Niemann-Pick Type C!

Personally this seems like a relief for so many families. One of the goals of the Niemann-Pick Children’s Fund is how can we directly impact families especially with this kind of disease. That of course is a huge task that seems daunting at times. There are so many other rare diseases out there that are equally devastating besides NPC. It is heart wrenching to know that there are so few options. Continue Reading