Joseph James Colton

Joseph James Colton
May 18, 2005 - May 19, 2010

It is with great sadness that we pass along news of the passing of little Joseph Colton, the son of James and Alexa Colton of Florida. Joseph James Colton, 5 years old, passed away on Wednesday, May 19th due to conditions resulting from Niemann- Pick Disease Type A/B.

Joseph was a happy child from the day he was born until the day he died. His enthusiastic smile brightened the room. Joseph loved to attend his brother's baseball and football games, watching and rooting for the teams to score.

Joseph enjoyed birthday parties, listening to music while dancing in his chair, hanging out in his community pool, taking morning or evening walks through his neighborhood and stopping to toss acorns into the water from the bridges.

We can all learn from Joseph's example how to embrace the simple pleasures available to us in each precious day. While his life was far too short, it was a life well-enjoyed and well-lived.

Our deepest sympathies go out to Joseph's family and friends in their loss.

For more information about Niemann-Pick Disease, please visit the NNPDF Web site.

Ironman Competitor to Raise Funds for Niemann-Pick Disease!

Ironman Mike Smith of Florida will compete in the Ford Ironman Triathlon in Coeur d'Alene (Idaho)on Sunday, June 27, to raise money and awareness for the National Niemann-Pick Disease Foundation.

The triathlon is the ultimate test of strength, endurance and determination, consisting of swimming 2.4 miles, biking 112 miles, and running 26.2 miles. An Ironman certainly embodies the NNPDF motto -- "Persevere!"

Mike's fundraising goal is $3,000 and he hopes to raise enough to qualify for a Janus matching fund donation, as well. Visit Mike's page to make a donation and read more about his motivation -- his vivacious wife, Annie OConnor-Smith, who is affected by Niemann-Pick Disease Type B.

Visit the National Niemann-Pick Disease Foundation's Web site to learn more about Niemann-Pick Disease and how you can help advance our Quest for a Cure!

FDA grants “Orphan Drug Designation” to Cyclodextrin for treatment of Niemann-Pick Disease Type C

Due to the efforts of Hugh and Chris Hempel, Dr. Caroline Hastings and Ron Browne, an application to the Food and Drug Administration requesting “Orphan Drug Status” for cyclodextrin has been approved.

What exactly does that mean for our NPD community?

It is important to emphasize that an Orphan designation does not make any assessment at all on how the drug works in clinical trials, whether it is safe or effective in patients, nor whether it will ever be commercially available – the Orphan designation’s main purpose is to make the development of the drug more financially viable for the developer.

Please visit the NNPDF's cyclodextrin page for a bit of background and glossary terms that will help you to understand this latest development.

18th Annual NNPDF Family Support and Medical Conference

Register now for the National Niemann-Pick Disease Foundation's 18th Annual Family Support and Medical Conference, scheduled for Thursday, August 5th through Sunday, August 8th, 2010!

This year's conference will be held in Toronto, Ontario, Canada, co-hosted by our sister chapter, the Canadian Chapter of the National Niemann-Pick Disease Foundation.

The NNPDF Family Conference is your best opportunity to learn the latest information about Niemann-Pick Disease directly from the eminent experts in the field, including physicians, researchers and scientists.

You will also meet, make friends, and have fun with many other families who are facing the challenges of NPD. We promise, you will remember forever the family connections you make at the Family Conference.

Family Conference registration packets were mailed out to NNPDF and CCNNPDF members last week. If you did not receive a packet, contact the NNPDF Central Office, or access the registration packet via the Family Conference page of the NNPDF Web site.

The Family Conference page is chock-full of information about the conference, including a conference schedule-at-a-glance, international travel tips, hotel reservation info, etc.

The deadline for registration is July 1st, so don't delay! Mark your calendar for Toronto, and start making plans to attend.

"Promising Therapies for Niemann-Pick Type C Disease"
NIH (NINDS) to Sponsor Meeting
June 3 - 4, 2010

The National Institutes of Health (NIH) National Institute of Neurological Disorders and Stroke (NINDS) is sponsoring a meeting in early June titled "Promising Therapies for Niemann-Pick Disease Type C Disease."

This meeting, set for June 3 - 4, at the Rockville, Maryland Hilton Hotel, is open to scientists, physicians and families until the maximum registration has been reached.

For more information, including the tentative agenda, travel and lodging information and a link to register, please visit our NewsLine page.

23-Month-Old Succumbs to Niemann-Pick Disease Type A/B

Caileen Harley Harrison (NPA/B)
May 7, 2008 - April 19, 2010

We are very saddened to pass along word of the death of little Caileen Harley Harrison, just short of her second birthday, due to complications of Niemann-Pick Disease Type A/B. Caileen was the daughter of NNPDF member Valerie Montgomery and Collis Harrison of California.

Kelly Puente of the Long Beach California Press-Telegram filed a story earlier this week about Valerie and Caileen. Caileen brought her family great joy despite her struggle with Niemann-Pick Disease. Visit the NNPDF NewsLine for a link to the Press-Telegram story and more photos.

Our deepest sympathy goes out to all Caileen's family and friends in their loss.

[photo credit: Brittany Murray/Press-Telegram]

Join us in Toronto August 5 - 8, 2010!

Mark your calendar and start planning your trip! The National Niemann-Pick Disease Foundation's 18th Annual Family Support and Medical Conference will be held in Toronto, Ontario, August 5th - 8th, and we want YOU to join us there!

We are so excited about this first-ever international conference, co-hosted by our sister chapter, the Canadian Chapter of the National Niemann-Pick Disease Foundation (CCNNPDF). We have already heard from several families from other countries that they plan to join us for this conference, and we expect an excellent turnout from the NPD scientific community.

We are planning a conference chock-full of information, inspiration and fun for families affected by Niemann-Pick Disease. This is your opportunity to hear the latest in NPD research directly from the eminent doctors, scientists and researchers in the field. You will have ample opportunities to meet and network with other families facing similar challenges, and you will take home friendships and memories that will last a lifetime!

Visit our Web page for all the details about our conference, travel tips, a link for hotel reservations, conference registration forms, and more. Be sure to check back often, as we are updating conference information almost daily.

Complete registration packets will also be mailed to all NNPDF and CCNNPDF member families in May, so be sure to let us know if your mailing address has changed.

Conference attendees from outside Canada, including the U.S., will need passports, so dig yours out and make sure it is still valid, or go apply for one ASAP, and start making your travel arrangements today. There are some great airfares out there, and for some, the train or bus may be even more reasonable options.

Please contact us if you have any questions about this year's Niemann-Pick Disease Family Support and Medical Conference, and we hope to see you in Toronto!