Passion To Help

For well over 30 years, Matthew has been on a quest to educate the public. At times we may think he is over the top but he has went up and beyond what most of us would do to fulfill not only our passion but to get people to notice him. I have to say that is admiring. We face the same challenge with a disease like Niemann-Pick Type C. It is rare and not a lot of people are aware. So we need to bring awareness to the matter to further educate about this rare disease that could open the doors to other well known diseases that plague our society. Our Vegas Casino Night is one of our attempts to draw the Kansas City community into a night of fun but equally important generating knowledge to the public.

Matthew will be presenting attendees with his American Benefit Plan (Value of $350) as a courtesy gift. He truly likes to give back.

What is Niemann-Pick Type C?

Niemann-Pick Type C is a genetic disorder that affects the cells ability to metabolize cholesterol. It is extremely rare and consequently always fatal. Although the age of death varies, it generally strikes in adolescents and childhood. To learn more visit here: About Niemann-Pick Type C

PR release by Evans Media Group: http://www.npcfund.org/LeskoInTown

More Lesko: http://www.Lesko.com or http://questionmarkguy.blogspot.com/
Brisan and Parker: http://www.BriParDun.com

Related posts:

1. Vegas in Kansas City Casino Night Fundraiser 11-11-11
2. FDA says Hold Off on Zavesca (Miglustat) approval.
3. Midwest Classic Tickets fore Charity Event

Gavin and His Mother’s Heartbreaking Story

Kristen and Fernando Lopez have 3 children from Little Elm, Texas. Gavin there oldest son has Niemann-Pick Type C disease. Recently they were on the TV show The Doctors talking about their guilt about having a child with Niemann-Pick Type C. This is very normal to have these feelings because we as parents love our children so much that we want to fix them even with a rare disease. The show was focusing around Overcoming Guilt.

Gavin was a healthy baby boy and by the age of 2 he started having issues with his speech, muscle’s going limp, and worrisome changes that had them concerned. For about a year they searched on what may be the reasoning behind it all. Kristin came across The Doctor’s TV show about Addi & Cassi Hempel who also has Niemann-Pick Type C disease. She felt that this was exactly the diagnosis she was seeking. After several tests and the whole process it was confirmed…Niemann-Pick Type C disease. For many this is a death sentence that no parent wants to here. Learn more by clicking here about Niemann-Pick Type C disease.

Here is the intro about the Lopez’s story:

How Niemann-Pick Disease Type C Affects the Body

E.R. physician Dr. Travis Stork explains how Niemann-Pick Type C affects the body.

Words of Hope for Niemann-Pick Type C Patients

Chris Hempel sharing words of hope for all patients and family currently coping with Niemann-Pick Type C.

Related posts:

1. New therapies – Niemann-Pick type C disease
2. Video: What is Niemann-Pick Type C disease? Can you share?
3. Promising Therapies for Niemann-Pick Type C Disease

Please participate on 10-10-10 for a $10 donation online to the NNPDF. They are truly doing some great things and we are thankful to be able to have them around.

Take the NPD 10 – 10 – 10  Challenge!
Please Help Raise Awareness for Niemann-Pick Disease

Intriguing dates such as 10/10/10 don’t come around all that often, and it seems fitting to proactively observe this once-in-a-lifetime occasion, especially since it falls during

October National Niemann-Pick Disease Awareness Month!

Participating in the NNPDF Central Office Challenge and showing support for the many families coping with Niemann-Pick Disease is one simple way you can mark the day by making a positive difference.   With just a few minutes of your time, you can help raise awareness and funding for all whose lives are affected and cut short by Niemann-Pick Disease.

Our 10-10-10 Challenge is to get 300 people to share information about Niemann-Pick Disease with 10 other people, via email, Facebook, Twitter, or other means (even via the hospitable old-fashioned way, face-to-face!).

Spreading the word about this rare disease is key to raising the essential funding for research which will find the treatments and cure we all work fervently toward, and to providing important family services for those who rely on us.

Here are some simple ways you can help families affected by Niemann-Pick Disease on 10-10-10:

1. Create awareness of NPD by forwarding this message to 10 (or more) people in your email address book.
2. Post this message (or your own message about Niemann-Pick Disease) to your Facebook page or blog.  Post a link to the NPD Awareness Facebook page (click the Facebook icon on the NNPDF Home page at www.nnpdf.org).
3. “Tweet” about NPD and share the NNPDF Web site address:  www.nnpdf.org
4. Take a group of friends out for coffee or dessert.  Tell them about NPD and the devastating effect it has on families.  (We will be happy to supply newly updated brochures for your use, just contact us.)
5. Donate $10 for NPD on 10-10-10 and encourage your friends and family to do the same.  Most of us spend at least $10 a week on discretionary items (snacks, soda, a movie, fast food).  Ten dollars doesn’t seem like a whole lot of money, but when many people each give ten bucks, it adds up!  Donations may be mailed to the address above, or made online via our Web site:  www.nnpdf.org.
6. Encourage 10 contacts to check out our NNPDF October Awareness Online Auction!  Bid high, bid often!  http://www.biddingforgood.com/nnpdf/2010
7. Get a “leg up” on your holiday shopping by purchasing ten of the darling new 2011 Puppies Calendars, “Treasuring Each Day.”  These calendars raise funds for the NNPDF’s essential research program and important Family Support Services.  For more details and to order, click here.

Thank you very much for your help and continued generosity, on 10-10-10 and always!
Those who live with the challenges of Niemann-Pick Disease need and truly appreciate your kindness and support, today and until the cure is found.  Together, we will Persevere in Our Quest for a Cure!

Together, we can make a difference!

Related posts:

1. New therapies – Niemann-Pick type C disease
2. Promising Therapies for Niemann-Pick Type C Disease
3. Canada Receives First Approved Treatment For Niemann-Pick Type C

Watch the video below and please share!

Summary edit of the Hollie and Annie films from sue lowe on Vimeo.

Related posts:

1. New therapies – Niemann-Pick type C disease
2. What is Niemann-Pick Type C?
3. MyDunkTank.com Dare By Steve Hugunin | Niemann-Pick Type C

Michael & Jennifer Stults 1-3-10

I’m very honored that me and my wife have been selected as one of the 30 people under the age of 30 who have contributed to our community in some manor. Although we feel we have a ton of work to accomplish, the Niemann-Pick Children’s Fund, Inc is dear to our heart. Our two oldest son’s Brisan and Parker Stults were diagnosed with a rare FATAL neurological disease called Niemann-Pick Type C Disease. Sometimes referred to as the “Childhood Alzheimer’s” because of the similar pathological features they share. They only have a childhood to live a lifetime.

Brisan and Parker heading to Disney World June 2009

Roughly 75 patients in the US currently and only 500 cases diagnosed worldwide EVER. This is a genetic disease on Chromosome 18. You get a Gene from your mommy and daddy. We each passed  a bad copy of our Gene which activates this horrible disease. The statistical odds of this situation coming to fruition is beyond comprehension.  In NPC it is the cells inability to metabolize cholesterol. Essentially the recycling factor breaks down and this good cholesterol your body needs to form cells ends up becoming the culprit. It stores all these fatty lipids in the Brian, Liver, Spleen, and Bone Marrow causing a domino affect of neurological problems that eventually leads to their premature death.

Currently there is no cure or effective treatment to date. With the help of showing the correlation with other common diseases like Arteriosclerosis, Stroke, Adult Onset Alzheimer’s, and even HIV/ Aids we can attract more researchers from these more profitable diseases into our tiny world. As many of you know most pharmaceutical companies wouldn’t want to invest in a small population that isn’t going to return their investment from the labs. It’s just a stark reality and capitalism.

VOTE ONLINE HERE  [ a $10 donation is required but…it ALL goes to Charity!

VOTE ONLINE HERE  [ a $10 donation is required but…it ALL goes to Charity!

We’re asking that you come out and support us. Tickets will be sold online at 30 Under Thirty or by visiting their website: http://www.connectinkc.com/Our%20Events and click on “Register” on this event. On September 10th, 2010 they we will recognize the candidates and their contributions to Kansas City. The event will be held at LUNA7pm-11pm. (www.lunakc.com) 1520 Grand Blvd Kansas City, Mo 64108. On this evening come out and vote for your favorite candidate (Hoping that is us!), enjoy catered appetizers, great drink specials (Including an hour of sponsored drinks), and dance the night away. Raffles & Prizes included. The candidate with the most votes that evening will have the proceeds of the event donated to their favorite charity of choice!! from

Thank you for your support!

Related posts:

1. UPDATE 2-US FDA panel backs new use for Actelion drug [Zavesca]

In Rockville, MD on June 3-4th many of the researchers looking into different areas and avenues for Niemann-Pick Type C came together to discuss promising therapies. Although research is making progress, parents of NPC patients never feel that research is happening fast enough. That is greatly to be expected. The NPC community is  making large strides in the right direction!

The National Niemann Pick Disease Foundation was very kind in putting together a recap of that meeting in a pdf format which is viewable here: Promising Therapies for Niemann-Pick Type C Disease.

Related posts:

1. New therapies – Niemann-Pick type C disease
2. What is Niemann-Pick Type C?
3. Canada Receives First Approved Treatment For Niemann-Pick Type C

Related posts:

1. New therapies – Niemann-Pick type C disease
2. Canada Receives First Approved Treatment For Niemann-Pick Type C
3. Why refer to it as the “Childhood Alzheimer’s”?

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.

Whether it is Niemann-Pick Type C Disease (NPC) or Alzheimer’s, they are extremely saddening to experience as a loved one. One affects mostly older adults and the other strikes predominantly in adolescents. Regardless, a main symptom in each disease is progressive Dementia along with similar pathological features that include the loss of intellectual function.

The question seems to arise often regarding why would you refer to Niemann-Pick Type C Disease (NPC) as the “Childhood Alzheimer’s” when they are technically two separate diseases? Touche’, great point Dr. Watson. My wife Jennifer absolutely hates when the two are referred to one another. I on the other hand am absolutely fine with the comparison and title.

When you tell someone about NPC, they kind of give you that “deer in the headlights” look along with the “side head nod” like a puppy. For any of you in sales or marketing the first thing you are taught is to make an impact statement to gain attention or some type of understanding of what your speaking about. When you say the “Childhood Alzheimer’s” people can instantly relate to someone or to a situation where they’ve heard about Alzheimer’s or Dementia.

Overall if you explain in a way tha t they are two separate diseases that share some similarities, you should be able to paint a mental picture for your audience. In no way do I feel by using this reference that it confuses nor threatens anyone. Just explaining what NPC is, is confusing enough. How can you make it any more confusing for people? Seriously.

You as a parent are the biggest advocate for your children. We need to let people know about this horrible disease. I believe it holds keys to other diseases affected by the storage of cholesterol in cells. Please visit our website www.NPCFund.org or my family’s personal page www.BriParDun.com.

Photo used under Creative Commons from Sharron Goodyear.

Related posts:

1. Dementia in Our Kids – The Uninvited Thief
2. Why Rare Diseases OUR Important
3. FDA says Hold Off on Zavesca (Miglustat) approval.

Serge Melancon, M.D., Montreal Children Hospital, commented: “Health Canada’s approval of Zavesca(R) (Miglustat) for the treatment of Niemann-Pick Type C disease is a wise choice and is very good news for patients and their families. Until now, no drug was effective against the neurologic manifestations associated with this debilitating familial disease. Canadian physicians will be without a doubt happy to have this therapy, which is already recognized in Europe and elsewhere around the world, to relieve their patients’ symptoms. As a clinical geneticist who has been dedicated to treating rare hereditary diseases for several years now, it is my fervent hope that the provinces will follow Health Canada’s lead and facilitate access to Zavesca(R) for patients who have already been diagnosed with this disease.”

Zavesca has shown some hope of relieving to a certain degree symptoms of Niemann-Pick Type C (NPC) and it’s devastating effects on families loved ones.  As the United States still isn’t completely convinced that it is ready to receive the FDA’s seal of approval, 75% of US families affected by NPC are not able to participate in the drug’s potential benefits due to non-FDA approval.

Jean-Paul Clozel, M.D. and Chief Executive Officer of Actelion commented: “I am very proud that Actelion – together with the scientific community – has been able to demonstrate the role of Zavesca(R) in reducing the progression of clinically relevant neurological symptoms in patients with NP-C. I would like to thank both the patients and their families who, over the years, have been involved in our clinical program with so much dedication, as well as all the clinical experts for their continuous support. Actelion will continue to support the rare disease community in its efforts to advance science and medicine for the patient.”

Overall this is great news and exciting in so many ways. All though this is a small step in the right direction, it is important to note that the ultimate cure or treatment for Niemann-Pick Type C (NPC) will possibly be a “cocktail” of remedies to treat its multiple symptoms.  Families must keep hope alive in their communities, government, and hearts that by persistent in many different facets that one day a rare disease like Niemann-Pick Type C (NPC) will open up doors to many other common diseases.

Persistence, it’s contagious.

Press Release Canadian Approval of Zavesca March 2010

Additional Information Regarding This Topic:

1. http://www.benzinga.com/pressreleases/m187674/zavesca-r-miglustat-first-treatment-available-in-canada-for-rare-progressive-n
2. http://www.marketwire.com/press-release/Zavesca-Miglustat-First-Treatment-Available-Canada-Rare-Progressive-Niemann-Pick-Type-Swiss-ATLN-1136457.htm
3. http://finance.alphatrade.com/story/2010-03-23/CCN/201003231315CCNMATHWCANADAPR_0594289001.html
4. http://www.nnpdf.org/npdisease_09.html

Related posts:

1. New therapies – Niemann-Pick type C disease
2. FDA says Hold Off on Zavesca (Miglustat) approval.
3. Historic FDA related drug advisory committee will meet | Zavesca

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?

I recently read a post from another family who has twin girls with Niemann-Pick Type C (Addi & Cassi Hempel) and it prompted me to finally put some of my thoughts down on “paper”. I agreed so much with what was wrote on that blog post that I couldn’t wait any longer. Niemann-Pick Type C is just one of many diseases that dementia plays a critical role in the quality of life that children/ young adults will have. I think about our caregivers, teachers at school, and even our children’s peers that are uneducated about dementia who could benefit from understanding the signs. They would be more equipped to cope and manage someone with dementia, especially our precious little ones who we love so much.

“Our lives are made in these small hours, these little wonders, these twists and turns of fate. Time falls away, but these small hours still remain.”

-Rob Thomas-musician

Dementia causes a loss of memory, confusion, issues with speech and language, changes in mood or behavior, loss of building new skills, losing their appetite and having the ability to complete simple tasks. Being a parent of Brisan and Parker Stults who has Niemann-Pick Type C, you learn to deal with a lot of patience. It’s not an easy task but with education you can better equip yourself to handle this additional burden. For instance with Parker we notice restlessness, unable to sleep at night, and very repetitive with phrases or just words. To most parents that can become pretty annoying!   The underlying issue is due to dementia but backed by their horrible disease Niemann-Pick Type C. It’s such a sad reminder that they only have a childhood to live a lifetime yet they’ll never remember it.

I read a website recently that said “dementia doesn’t happen to children”. That is false because of the stigma placed with this topic. Dementia is an uninvited thief who holds nothing back. Just remember children can have dementia to and this isn’t just an elderly issue. Find the education you need to bring awareness to your community about this issue.

Dementia in children, teenagers and young adults A guide for parents, teachers and care professionals

Related posts:

1. Dementia. It changes your life.
2. Why Rare Diseases OUR Important
3. Social Security Adds 38 New Medical Conditions that Qualify for Disability