Spring/Summer is Fundraising and Awareness Time!

The busy spring and summer fundraising and awareness-raising season is now in full swing! Warmer weather is peak time for outdoor events, festivals, community gatherings, and other opportunities to advance the mission and vision of the National Niemann-Pick Disease Foundation, on behalf of all affected by NPD.

Check out our Upcoming Events and Recent Events pages for a taste of all the great activities going on around the country in support of Niemann-Pick Disease Research and NNPDF Family Services programs.

As just one example, the photo above shows the motorcycle being taken around to various events in Florida, to be raffled off to raise funds in memory of little Ryan Richardson (NPC).

If you've never done any fundraising, or if it's been a few years since you held an event, now is the time! Check out the postdoctoral fellowship research projects the NNPDF is currently supporting for some motivation, and let us help you get started in your community.

We can provide brochures (ASMD Type A/B brochures or Type C brochures, all updated in 2010), newsletters and (NEW!) posters for your fundraising needs.

The ever-popular periwinkle PERSEVERE wristbands are an easy way to raise funds and awareness. Packaged individually, each wristband contains a message of appreciation for supporting our Quest for a Cure. Wristbands are $1.00 each and can be sold with minimal effort at any event, or at the checkout counters of supportive local businesses.

One of our member families makes colorful, eye-catching vinyl banners to bring attention to your booth or event. And we can help you with ideas, best practices, press releases, or with requests for official proclamations.

Thank You to all the families, extended families and community members who support the important work of the NNPDF! Visit our NewsLine page for more details about the above ideas, and please let us know how we can be of assistance.

NNPDF Research Updates

Postdoctoral Fellows Report on their Work

The NNPDF is very pleased to currently be providing support to four postdoctoral research fellows, all conducting studies on various aspects of Niemann-Pick Disease.

The four fellows, Dr. Fabrizio Vacca, Dr. Ian Williams, Dr. Nicholas Cianciola, and Dr. Dorothea Maetzel, recently sent us updates on their ongoing research projects. The NNPDF extends its great appreciation to these scientists as they work to unlock the mysteries of NPD, and to hasten the journey to effective treatments and a cure.

Please visit the NNPDF’s Fellowships Funded page to read the latest reports from our postdoctoral fellows.

Research funded by the NNPDF is made possible largely due to the diligent efforts of our member families and their extended support networks hosting local community events. The NNPDF is truly grateful for this support! Thank you for your help as we PERSEVERE in our Quest for a Cure!

Time is Short - Make a Year-End Gift Today

Two Days Left to Make a Tax-Deductible Contribution

When you make a financial donation to the National Niemann-Pick Disease Foundation, you get so much more than a tax deduction. You will enjoy the wonderful feeling of knowing you’re making a tangible difference in the lives of children, adults and families living with the challenges of NPD!

Your gift drives the essential research that will unlock the mysteries of NPD, bringing effective treatments and therapies for the ravages it wreaks on the body and brain.

Your generosity also provides important support services for the families who walk the lonely road of a rare disease, helping them connect with others who know and understand, and providing them with the information they so desperately need.

Even the most modest donation can make an enormous difference when added together with the gifts of others. Please, remember the families of the NNPDF in your year-end charitable giving!

Thank you for making a difference in the lives of those battling Niemann-Pick Disease!

Make a donation to the NNPDF

Holiday Gifts & Year-End Giving to Support NNPDF

Programs of Research and Family Services Benefit

Online Shoppers: Use this special Amazon.com link to shop online, and GoodSearch when you surf the Web – both benefit the NNPDF-- and you can receive special coupons and discounts, as well.

Each year, a growing number of families depend on the NNPDF for medical information, research updates and emotional support as they struggle to learn about a rare disease while caring for an ill family member.

We, in turn, depend on those with the compassion and the financial resources to help us drive forward the research that will ultimately result in treatments and a cure for NPD, and to provide support services to those affected by this disease.

As you consider your year-end tax planning, we’d like to remind you that a gift to the NNPDF can reduce your income tax obligation, while providing meaningful support for the work of the foundation. If you designate your gift in honor of a loved one, the NNPDF will gladly send them an acknowledgement card.

The NNPDF gratefully accepts direct financial donations, United Way and Combined Federal Campaign contributions, gifts of stock, in-kind donations, and planned gifts such as bequests, beneficiary designations, or life insurance policies. Please visit our special page for more information about these philanthropic year-end gift ideas.

Donations may be mailed to: NNPDF; P.O. Box 49; Fort Atkinson, WI, 53538. Online credit card donations may be made here. We invite you to contact the NNPDF Central Office with any questions.

If you have friends or relatives who might be interested in making a year-end gift in honor of a loved one, please forward this information to them, as well.

2011 Wall Calendars and PERSEVERE Clothing
Ring in 2011 with these quality 2011 wall calendars in two very special designs! Choose from playful puppies or lush scenic paintings by New England Artist Don Greer, grandpa of 7-year-old Naomi Tyrrell (NPC). Calendars are just $10 each, and shipping is free on quantities of five or more.

Encourage your family and friends to “Press On! Keep At It! Get it Done!” with the very popular, stylish and comfortable PERSEVERE wear t-shirts and hoodies.

Sales of these calendars and PERSEVERE wear benefits the NNPDF, raising funds for research and family support services.

Order calendars and PERSEVERE wear directly from Lorna Tyrrell – click the links above for complete details and order forms.

Thank you for your generous support, and "Happy Holidays" to you!

One Child's Journey through Niemann-Pick Disease

One Child’s Story Through Niemann-Pick Disease Type C

Stacey Lynne Vorpahl (NPC)
January 3, 1985 – October 9, 2004

In loving memory of Stacey and all the other precious children lost to NPD, and in honor of those still struggling, please join with us in our Quest for a Cure. Follow the link to view Stacey's story, as told by her parents in text and photos.

10 – 10 – 10 Challenge
Please Help Raise Awareness for Niemann-Pick Disease

Meet the NNPDF's 10-10-10 Challenge and take at least one simple action on October 10,2010, to spread the word about this devastating disease.

Thank you for your help and support, on 10-10-10 and always!

2011 Puppies Calendar Now Available!

Get ready for 2011 with the latest edition of the darling Puppies Calendar, "Treasuring Each Day!"

Featuring playful puppies and sayings of inspiration and hope, this very special wall calendar is dedicated to the children and families who struggle every day with the challenges of Niemann-Pick Disease (NPD).

Get a jump on your holiday shopping by ordering ten calendars on 10-10-10, supporting the NNPDF's program of research and important Family Support Services at the same time!

Your purchase of these calendars gives hope to families affected by NPD as funds are raised for essential research into treatments and a cure. Turning the page each month, we are reminded that we are in a race against time to find treatments and a cure for all children and adults with NPD.

NNPDF member Lorna Tyrrell created the Puppies Calendars in honor of their seven-year-old daughter (who loves puppies and bunnies and all things cuddly!), Naomi (NPC). Please order calendars directly from the Tyrrells using the order form available here.

Thank you for your support of all those affected by Niemann-Pick Disease, on 10-10-10 and every day!

Take the 10-10-10 Niemann-Pick Disease Challenge!

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. Please, will you help us meet our goal? Click here for six easy ways you can help us meet this October Awareness Month challenge!

One decidedly FUN way to participate in October Niemann-Pick Disease Awareness Month is to shop the Online Auction! Bidding is now open! Bid high, bid often!

It's also not too late to donate items to the auction. Check out this page for more details, or contact the NNPDF Central Office.

As always, Thank You most sincerely for the continued love and support you give the families who deal with the challenges and heartbreak of Niemann-Pick Disease!

FDA Grants Clearance for Experimental Intrathecal Administration of Cyclodextrin

The FDA has granted clearance of an Investigational New Drug (IND) application for administration of Hydroxypropyl Beta Cyclodextrin (cyclodextrin) into the central nervous systems of two patients with Niemann-Pick Disease Type C.

The cyclodextrin will be given intrathecally (into the space under the arachnoid membrane of the brain or spinal cord), first via lumbar injection, and then into the brain’s ventricle system. Children’s Hospital & Research Center of Oakland, California, issued a press release, which can be accessed from the NNPDF's NewsLine page.

The NNPDF would like to note the following regarding cyclodextrin:

1. Cyclodextrin has shown promise as a potential therapeutic in animal trials. However, issues of toxicity have also arisen.
2. The NNPDF is aware that cyclodextrin is being used on a single-patient Investigational New Drug (IND) basis in the U.S. and in Brazil.
3. More research is needed to understand issues related to drug delivery, efficacy and safety.
4. The NNPDF will keep the NPC community apprised of developments regarding cyclodextrin.

Amy Dockser Marcus published this related article in her Wall Street Journal Health Blog on September 23.

For more information about cyclodextrin, cyclodextrin's orphan drug designation, a glossary of terms, and links to the FDA, please visit our cyclodextrin page.

Cassandra Phillipa Stallard

Cassandra Phillipa Stallard (NPC)
May 30, 1989 - September 21, 2010



We are very sad to learn of the death of 21-year-old Cassie Stallard due to the effects of Niemann-Pick Disease Type C (NPC).

According to Cassie's mom, Trish, Cassie died peacefully in her sleep after a day enjoyed strolling on the beach and a trip to a favorite coffee house.

All our sympathy to the Stallards and their extended family and friends in their loss.

To learn more about Niemann-Pick Disease, please visit the National Niemann-Pick Disease Foundation Web site.

October is Niemann-Pick Disease Awareness Month!

October is the time to raise awareness about Niemann-Pick Disease!

We need to get the word out about this rare disease to doctors, families, government leaders and the general public.

We invite and encourage each family to look within their family structure, friends and community support networks to create their own team and sponsor an awareness campaign and/or fundraising event during October 2010.

The NNPDF has a wealth of experience and resources to support you in any fundraising/awareness project you'd like to undertake, from a simple "Persevere" wristband sale or a "Family and Friends" letter, up to an all-out gala or golf tournament. Take a look at what other NNPDF families are cooking up for October.

One easy and effective way to support the foundation's goals of research into treatments and a cure, and to support families affected by Niemann-Pick Disease, is to participate in this October's Online Auction as a donor and/or a buyer.

It’s a win-win-win situation – the winning bidder gets a great deal on something they like with the added satisfaction of knowing their money is supporting a favorite cause, while the donor has a great feeling knowing their gift will go far to help families affected by NPD. Best of all, the NNPDF is able to use the proceeds to diligently advance our mutual Quest for a Cure!

There are three ways you can help us make this auction a big success: donate an item(s) for auction; bid on auction items; and spread the word to all your family and friends so they can bid, too! (Please visit our Web site for all the details.)

We are busy getting our auction site set up so you can start to "window shop" the items up for sale. Please check the foundation's Online Auction Web page next week for a link to the auction itself. And we hope to hear from you soon regarding what you might be able to donate for this exciting new fundraising opportunity!

As always, Thank You for your continued support! Together, we can make a difference!

Sarah Elisabeth Glassman

Sarah Elisabeth Glassman (NPA/B)
Jan. 30, 2007 - Aug. 30, 2010

With great sadness we pass along word of the death of Sarah Elisabeth Glassman, age 3 years, from the effects of Niemann-Pick Disease Type A/B (ASMD).

Sarah is survived by her parents, Aaron and Valerie Glassman of Norfolk, Virginia; her brother, Zachary; grandparents Dr. Myron and Nanci Glassman and Brenda Bowling; and many aunts, uncles, cousins, friends and admirers.

"Loved by all who knew her, delicate and tiny Sarah was an amazing inspiration. It was an honor to be with this little angel. She taught everyone the true value of each day on earth and gave us all so many happy memories."

In lieu of flowers, memorials can be made to the Edmarc Hospice for Children (www.edmarc.org) or the National Niemann-Pick Disease Foundation.

Carnival Raises Funds in Memory of Karen Sullivan

Jason, Sarah and Abby Sullivan, ages 10, 7 and 5, respectively, organized and held a backyard carnival to raise funds for the NNPDF in memory of their aunt, Karen Sullivan, who died in 2004 as a result of Niemann-Pick Disease Type C (NPC).

After several weeks of planning, the children made signs and posters to advertise and invite their friends and neighbors to come for games, prizes, and food.

The whole family got involved as one grandma made funnel cakes and another ran the bean bag toss, while the two grandpas ran the golf game and served as the event photographer. The late-summer heat wave even broke in time for a perfect evening, and the neighborhood was very supportive, raising almost $500 for the fight against NPD!

Jason, Sarah and Abby are the children of Kevin and Jean Sullivan and the grandkids of Art and Nancy Sullivan of Chesterfield, Missouri. Nancy recently retired from the NNPDF Board of Directors and Art serves on the NNPDF's Finance Committee.

Thank you, Jason, Sarah and Abby, and the entire Sullivan family!

To see more photos from the carnival, and to read about other family fundraisers advancing the Quest for a Cure, visit the NNPDF's Upcoming Events page.

Princess Monica Celebrates First Birthday!

The 18th Annual National Niemann-Pick Disease Family Support and Medical Conference was held August 5th through 8th, in Toronto, Canada.

One of many highlights of the conference was the "Princess Party" held in honor of the first birthday of Monica Taillefer, daughter of Heather Patenaude-Taillefer and Simon Taillefer of Quebec.

The party was complete with a tiara for Princess Monica, crowns for all the kids, cake, of course, and special appearances by Cinderella and Snow White.

For more conference details and a link to see dozens of great photos from the event, visit the NNPDF NewsLine page.

Promising Therapies for NPC

"Promising Therapies for Niemann-Pick Type C Disease"
NINDS Meeting
June 2010

We have had quite a bit of discussion on our listserv pertaining to the recent research and treatment options for Niemann-Pick Type C (NPC) Disease.

To provide some background and insight for these continuing discussions, we have compiled a summary of the presentation abstracts from the "Promising Therapies for Niemann-Pick Type C Disease" meeting sponsored by the National Institute of Neurological Disorders in Rockville, Maryland, June 3-4.

Visit the NNPDF's Latest Research page to read the presentation abstracts from this important conference.

Emily Jane Hrbacek

Emily Jane Hrbacek
July 15, 1999 - July 1, 2010

It is with great sadness that we pass along word of the loss of Emily Jane Hrbacek due to Niemann-Pick Disease Type C. The beloved daughter of Scott and Laura Hrbacek of Eureka, Missouri, Emily was just shy of her 11th birthday.

We received this message from Emily's family:

This morning Emily passed away peacefully and is no longer suffering. Emily passed from the complications of Niemann-Pick Disease. Emily put up a long fight but in the end now the fight is over. Emily had the most beautiful eyes and a smile that melted everyone's heart. Emily was the best child that God could have given us and she will be missed by many. God bless our angel.

A visitation will be held at Most Sacred Heart Church, 350 East Fourth Street, Eureka, on Sunday, July 4th, from 3:00 - 8:00 p.m. Funeral mass will be at 10:00 a.m. on Monday, July 5th, also at Most Sacred Heart, with burial at Holy Cross Cemetery.

Our deepest sympathy to Emily's parents, family and many friends.

To learn more about Niemann-Pick Disease, visit the National Niemann-Pick Disease Foundation (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!

"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!