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