From the Desk of Chris Klauer, NNPDF Family Services & Marketing Coordinator ~ Jan 15th, 2016

Here in Wisconsin we just finished with a cold snap and warmed up to 35 degrees today. However, another cold snap is just around the corner. One of my favorite photographers took the picture of the ice caves near Apostle Islands National Lakeshore. Thought you might like to see some of the beauty of Wisconsin winters.

© Copyright 2015, Life in the Northwoods

My "Family Support Services" update this week highlights:

- 24th Annual NNPDF Family Support and Medical Conference

- Now I Lay Me Down To Sleep

- Infinity Coaches’ Charity Challenge to raise money for NPC research

- CTD Press Release

Please feel free to contact me at: nnpdf@nnpdf.org or give me a call at: 1-877-287-3672 if you have any questions pertaining to the work and "Family Support Services" afforded to the members of the foundation. If you find a great resource, please pass it along so I can share it with the rest of our NNPDF Family Membership!

Have a great weekend!

Click here to view the full post: http://www.nnpdf.org/FamilyServiceSupport.html#Jan1516

Chris Klauer

NNPDF Family Support and Marketing Coordinator

Special Update from the NNPDF Board of Directors ~ Year-End 2015

Dear NNPDF Families and Friends,

As we wrap-up year-end 2015 and embark on all that 2016 has in store for the NNPDF, the Board of Directors wanted to provide you with a Finance Committee update. The Finance Committee is responsible for continually reviewing the financial affairs of the NNPDF during the year, preparing the budget, reviewing any anticipated expenses, and communicating the financial condition of the NNPDF to the Executive Committee and NNPDF Board of Directors. 

Please find a year-end 2015 update from the NNPDF Finance Committee Chair, Jill Flinton, of Charlton, New York. Jill serves on the foundation board in a volunteer capacity and in memory of her son, Daniel (NPC) who passed away from on November 11, 2011 at the tender age of four years.

To access the NNPDF Foundation Financials and Forms web page directly ~ simply go to the NNPDF Web Site Home Page at www.nnpdf.org, choose About Us, follow the link to the NNPDF Financial Files and Form to see most recent update post to the NNPDF membership; dated: Jan 12th, 2016 or follow this direct link: http://nnpdf.org/aboutus_02.html#FinCommittee2

If you should have any questions related to this information and/or update ~ please feel free to contact the foundation for assistance.

UPDATE: 2016 NNPDF Family Conference Date and Location Announced

The NNPDF is excited to announce that the 24th Annual Family Support & Medical Conference will be held in Boston, Massachusetts on August 11-14, 2016.

Specific details associated with this summer 2016 event are being finalized and will be posted as soon as they become available!

For now, please pencil in these dates on your summer calendar ~ and ~ we hope to see you all there!

From the Desk of Chris Klauer, NNPDF Family Services & Marketing Coordinator: January 8th, 2016

My update this week highlights:

• Family Voices
• Understanding Breakthrough Therapy Designations
• NPD “In the news!”

Please feel free to contact me at: nnpdf@nnpdf.org or give me a call at: 1-877-287-3672 if you have any questions pertaining to the work of the foundation. If you find a great resource, please pass it along so I can share it with the rest of our NNPDF Family Membership!

Click here to view the full post: http://www.nnpdf.org/FamilyServiceSupport.html#Jan082016

Have a great weekend!

Chris Klauer

NNPDF Family Support and Marketing Coordinator

Vtesse, Inc. Announces FDA’s Granting of Breakthrough Therapy Designation for VTS-270 (2-hydroxypropyl-β-cyclodextrin) in Niemann-Pick Type C1 Disease ~ Dateline: 01/06/2016

Vtesse, Inc. Announces FDA’s Granting of Breakthrough Therapy Designation for VTS-270 (2-hydroxypropyl-β-cyclodextrin) in Niemann-Pick Type C1 Disease ~ Dateline: 01/06/2016

The National Niemann-Pick Disease Foundation (NNPDF) is pleased to share with our NPC Community Members that the Food and Drug Administration (FDA) has granted VTS-270 (2-hydroxypropyl-β-cyclodextrin) a Breakthrough Therapy Designation. The FDA Breakthrough Therapy designation is designed to expedite the development and review of drugs within the FDA regulatory process.

This designation is granted by the FDA when the preliminary clinical data indicates that the drug may demonstrate substantial improvement on clinically significant endpoint(s). The Breakthrough Therapy designation is distinct from the FDA’s other mechanisms to expedite drug development and review, and will allow for a close collaboration between Vtesse and the FDA on the VTS-270 (2-hydroxypropyl-β-cyclodextrin) development program.

Both the FDA and the European Medicines Agency (EMA) had previously granted Orphan Drug status to VTS-270, which is currently in a pivotal Phase 2b/3 clinical trial. For additional information regarding VTS-270 (2-hydroxypropyl-β-cyclodextrin) clinical trial, visit Vtesse's website.

~ Recording & Slides of Dec. 14th Webinar Now Available ~

In addition to the above press release, the NNPDF Central Office recently obtained the Webinar recording and slides associated with the Vtesse “Town Hall” Webinar hosted for the NPC patient community on December 14th, 2015.

Click here to Read the Press Release dated Jan 6th, 2015: http://www.nnpdf.org/Newsline2015.html

We Will Persevere!

As 2015 draws to a close, families and friends will gather to celebrate all of the milestones afforded them throughout 2015. As we all take the time to reflect and close the last chapter of 2015, we all look ahead to the new chapters which await us in 2016 ~ there are new advances and milestones awaiting our patients and families!

Often, for our NPD families, the future is guided by the fear and trepidation of what awaits as the progression of Niemann-Pick Disease inevitably begins to take its toll on their loved ones. BUT ~ as we look ahead towards 2016 ~ it is with a glimmer of hope that the ongoing and upcoming clinical trials for a number of therapies into NPD will be successful in slowing the disease progression.

We ask that the members of the foundation draw strength from the prose of Helen Keller, “Your success and happiness lies in you. Resolve to keep happy, and your joy and you shall form an invincible host against difficulties.”

To cap off 2015, we have created a “Year-end” e-Newsletter to share with the NNPDF community.

It includes:

- Thanks for your ongoing financial support in 2015 ~ “Year-end” donation reminders!

- In Memoriam ~ Remembering Those We Lost in 2015

- Coping with Loss During the Holidays

- 2015 E-Newsletters Recap

- 2016 Annual NNPDF Board Meeting

Click here to view the 2015 End of Year e-Newsletter: http://www.graphicmail.com/new/viewnewsletter2.aspx?SiteID=4117&SID=0&NewsletterID=1350530

As the New Year approaches with hopes anew, the NNPDF wishes to extend to you and your family a wonderful year ahead. Happy New Year!

Giving the Gift of Hope!

During these tight fiscal times, we understand that there are many worthy causes vying for your donation dollars. You can be assured that your gift to the NNPDF will be greatly appreciated and faithfully stewarded to bring the greatest benefit to families dealing with Niemann-Pick Disease in 2016 and beyond!

Johnathan Spencer, age 9 ~ NPC ~ 

National Institutes of Health Phase 1 Clinical Trial Participant

Thank you in advance for your help towards our Quest for a Cure and supporting the National Niemann-Pick Disease Foundation (NNPDF) so that we can provide the gift of hope for our NPD patients and their families.

To find out how you can help give the Gift of Hope, click here for the e-Blast: http://www.graphicmail.com/new/viewnewsletter2.aspx?SiteID=4117&SID=0&NewsletterID=1350376

Season's Greetings from the NNPDF!

The National Niemann-Pick Disease Foundation would like to wish you all season's greetings! 

Whatever is beautiful, Whatever is meaningful, Whatever brings you happiness...May it be yours this Holiday Season.

Best wishes to all of our Niemann-Pick Disease Foundation members and their supporters. 

May the warmth of hearth and home fill your hearts with joy this holiday season! 

~ The NNPDF Office Staff and Members of the Board of Directors

NPC Vtesse Clinical Trial & Central Office Updates

Dateline: Dec 21st, 2015

Dear NNPDF Family & Friends,

The National Niemann-Pick Disease Foundation (NNPDF) is pleased to be able to share with our NPD community families updates associated with the following:

- Vtesse VTS-270 NPD Type C Clinical Trial UPDATES

- NNPDF Equipment Exchange Program and NEW posted items

Click here to view the NNPDF e-Blast: http://www.graphicmail.com/new/viewnewsletter2.aspx?SiteID=4117&SID=0&NewsletterID=1348668

Dec 18th, 2015 ~ From the Desk of Chris Klauer, NNPDF Family Services & Marketing Coordinator

Dear NNPDF Families and Friends,

The Holidays are coming! The Holidays are coming! No don’t run for the hills! Instead check out the Holiday Survival Guide that I found!

My update this week highlights:

- Holiday Survival Guide

- NNPDF & CCNNPDF Membership Update

Please feel free to contact me at: nnpdf@nnpdf.org or give me a call at: 1-877-287-3672 if you have any questions pertaining to the work of the foundation. If you find a great resource, please pass it along so I can share it with the rest of our NNPDF Family Membership!

Have a great weekend!

Click here to view this week's Family Services Update post: http://www.nnpdf.org/FamilyServiceSupport.html#Dec182015

Chris Klauer

NNPDF Family Support and Marketing Coordinator

Ghazi Tamim (NPA) 10/21/2013 - 12/07/2015

Dear NNPDF Families and Friends,

The NNPDF Central Office today received notice of the passing of Ghazi Taimur (10/21/2013 – 12/7/2015 ~ NPA).

Ghazi’s father Taimur writes: “We are saddened to inform all that our beloved son Ghazi, after 2 ½ years of fighting NPA died on 7th of December 2015. Please remember him in your prayers.”

To Ghazi’s loving parents, Taimur and Fatima please know that your NNPDF family is here for you and to let us know if you need anything in the coming days, weeks and months ahead. We are holding you all in our hearts and thoughts.

Year-end Shopping for the Gift of Hope!

Dear NNPDF Families and Friends,

As year-end approaches and shopping for loved ones begins to hit “fever-pitch” level….more and more of us are turning to on-line shopping services for assistance in finalizing our Santa wish list! If you have found yourself letting your “fingers do the shopping on-line” ~ we have an easy way in which your shopping efforts can benefit the NNPDF and our programs at the same time.

The NNPDF has taken steps this holiday season and throughout the entire year, to provide the opportunity for our supporters who enjoy on-lining shopping the ability to “Gift of Hope for a Brighter Future” for all those diagnosed with Niemann-Pick Disease (NPD). 

Click here to view the NNPDF e-Newsletter highlighting ways you can shop to support the Foundation: http://www.graphicmail.com/new/viewnewsletter2.aspx?SiteID=4117&SID=0&NewsletterID=1346905

ALERT: New Recruiting Sites for VTS-270 (2-hydroxypropyl-β-cyclodextrin) for NPC

ALERT: United States NPD Type C Families & Friends

The NNPDF central office noted today that additional investigational sites for Vtesse’s VTS-270 (2-hydroxypropyl-β-cyclodextrin) Phase 2b/3 clinical trial for NPC has been updated and is now actively recruiting patients at the approved clinical trial centers in Orange, CA & Allentown, PA.

Families should follow enrollment criteria as provided on the www.clinicaltrials.gov page. To view these criteria, location details and for additional contact information please visit www.clinicaltrials.gov and refer to study reference number: NCT02534844

For all previous updates regarding this clinical trial, visit the the links below:

Vtesse Phase 2b/3 Clinical Trial Historical Timeline can be viewed: http://www.nnpdf.org/Vtesse.html

NIH Phase 1 Clinical Trial Historical Timeline can be viewed: http://www.nnpdf.org/Cyclodextrin.html

 

Kaitlyn Bexton (NPC) ~ 01/24/2001 ~ 12/8/2015

Dear NNPDF Families and Friends,

It is with a heavy heart that we notify our NPD family membership of the passing of Kaitlyn Bexton (January 24, 2001 – December 8, 2015 ~ NPC), who courageously fought the NPC battle for 14 years. Kaitlyn loved watching “Dancing with the Stars”, listening to music and spending time with her family. Katie had an infectious smile and filled a room with her spirited, happy and playful energy. 

Kaitlyn’s family takes comfort in the fact that she is now playing with her sister Ally (Alexandra), who also suffered from Niemann-Pick Disease Type C and passed away in 2009 at the age of 10. 

To Kaitlyn’s loving parents, Stasha and Michael, and beloved younger sister, Samantha, please know that your NNPDF family is here for you and to let us know if you need anything in the coming days, weeks and months ahead. We are holding you all in our hearts and thoughts.

A link to Kaitlyn’s obituary will be posted as soon as it is available on the NNPDF website: http://www.nnpdf.org/Newsline2015.html#Bexton

 

Register for Webinar on VTS-270 (2-hydroxypropyl-β-cyclodextrin) for NPC ~ Dateline: 12/07/2015

Register: US and Canadian Audience, 12:30 PM EST*, December 14, 2015

https://attendee.gotowebinar.com/register/7538191617966914050

*9:30am Pacific, 10:30am Mountain, 11:30am Central, 12:30pm Eastern

Dear NNPDF Niemann-Pick Type C (NPC) Family Members,

The National Niemann-Pick Disease Foundation (NNPDF) is pleased to be able to share with you an opportunity to attend a town hall webinar hosted by Vtesse, Inc. with regards to the VTS-270 (2-hydroxypropyl-β-cyclodextrin) clinical trial for Niemann-Pick Disease, Type C (NPC).

Please join Vtesse on December 14, 2015 where Dr. Paul Gissen and Dr. Elizabeth Berry-Kravis will be providing an update on the clinical trial and will be available to address your questions. Vtesse is committed to keeping the community fully informed and soliciting feedback as they move through the clinical trial process.

Visit the Vtesse Clinical Trial page to read the full post and for more information on registering for the webinar: http://www.nnpdf.org/Vtesse.html#Dec072015

Sincerely,

Nadine M. Hill

Executive Director

National Niemann-Pick Disease Foundation

Vtesse Phase 2b/3 Clinical Trial Historical Timeline can be viewed: http://www.nnpdf.org/Vtesse.html

NIH Phase 1 Clinical Trial Historical Timeline can be viewed: http://www.nnpdf.org/Cyclodextrin.html