NPC Research Funding Update ~ 2014 Million Dollar Bike Ride

Dear NNPDF Friends and Families,

The NNPDF Central Offices are pleased to share with our NPC Community how the funds raised by Team NPC and the collective efforts of the Reedy-DiGiovani, Mayo, Coppola & Honsinger families have been applied!

Visit the NNPDF Newsline web page to see how the funds are being applied: http://www.nnpdf.org/Newsline2015.html#Million

Team NPC ~ your efforts made this possible and we want to thank you again for all the work you do to further NPC research! You are an inspiration on how to Persevere in our continued, collaborative Quest for a Cure! 

GO TEAM NPC!!!

$100,000 for NPC Research ~ Infiniti Coaches Charity Challenge - Round 2 ~ January 26th - February 15th, 2015

Dear NNPDF Families and Friends,

As of January 26th through February 15th, 2015~ Round 2 of the Infiniti Coaches' Charity Challenge is underway! Matt Painter, Purdue University coach, is representing the Smith Family BReaKthru Fund to win up to $100,000 for NPC research! 

Currently, Coach Painter is tied at 24% with another participant, so let's keep the momentum going for this round and into the remaining rounds. Remember to vote daily for Coach Painter and let's help get him to the $100,000 Champions spot this year!

Click Here to Vote for Matt Painter

Visit the NNPDF Newsline for additional information about the Challenge: http://www.nnpdf.org/Newsline2015.html#Infiniti   

Update from Genzyme on Acid Sphingomyelinase Deficiency (ASMD) Development Efforts Adult Trial ~ January 28th, 2015

Dear NNPDF Families and Friends,

The NNPDF is pleased to provide you with the following update from Genzyme, a Sanofi Company, on NEWLY released information about the adult Phase 2 / 3 clinical trial of recombinant human acid sphingomyelinase (rhASM).

Visit the NNPDF Genzyme web page to view the full announcement: http://www.nnpdf.org/npresearch_11.html#Jan282015

Please be advised of the following details associated with the adult Phase 2 / 3 clinical trial #: NCT02004691:

• The study is NOT yet open for patient recruitment
• Inclusion / Exclusion criteria are provided in the post
• Estimated enrollment of 35 adult patients
• MUST be 18 years and older
• The number of country clinical sites and locations has NOT yet been determined ~ but there will be clinic sites in multiple countries.

More information is detailed at the trial page link here:https://www.clinicaltrials.gov/ct2/show/record/NCT02004691?term=Niemann-Pick+Genzyme&rank=4

FDA Grants Orphan Drug Designation for Arimoclomol ~ Dateline: 01/27/2015

Dear NNPDF Families and Friends,

Orphazyme today announced that the Food and Drug Administration (FDA) Office of Orphan Products Development (OOPD) has granted an orphan drug designation for Orphazyme’s Arimoclomol as a new treatment for Niemann-Pick disease type C.

The US orphan drug designation complements the corresponding EMA orphan drug designation that was granted for the program in 2014.

“The recent successful completion of a €20m Series B financing round, together with the FDA and EMA orphan drug designations, establish a solid foundation for initiating our clinical studies in NP‑C with the purpose of providing arimoclomol as a safe and effective therapeutic option to patients suffering from this devastating disease as well as relief to their families,” says Dr. Carlos R. Camozzi, Chief Medical Officer of Orphazyme.

Click here to view the full press release: http://www.orphazyme.com/index.php/news/news-releases/53-fda-grants-orphan-drug-designation-for-arimoclomol

“The National Niemann-Pick Disease Foundation (NNPDF) does not engage in the practice of medicine. It is not a medical authority nor does it claim to have medical knowledge. This site is an educational service of the National Niemann-Pick Disease Foundation and is not meant to provide diagnostic or treatment advice. Information contained or suggested on this Web site does not constitute medical advice. For all information related to care, medication or treatment, the NNPDF recommends consulting a physician to determine if information presented is applicable. Please review these additional cautions (http://www.nnpdf.org/CyberCautions.html) about medical information provided on the Internet." 

Parker Maddox Stults ~ January 8th, 2006 ~ January 23rd, 2015

Dearest NNPDF Families and Friends,

It is with profound heartbreak that we advise our NPD membership community that Parker Maddox Stults, one of our brave and courageous Niemann-Pick type C children, lost his fight against NPC on January 23rd, 2015 at the tender age of 8 years. Parker is survived by his parents, Jennifer & Michael Stults, & by his younger brother Duncan. He was proceeded in death by his older brother Brisan less than a year ago, who passed on March 4th, 2014 from NPC at the age of 9 years. 

Parker's mother, Jennifer, wrote of her son on Facebook:

"Parker flew into the arms of Jesus and was welcomed into Heaven by his big bubbas Brisan. Many that loved him were here to comfort him and love on Michael, Duncan and myself. Our two precious boys are together once again." 

Please visit the Newsline for the family memorial service information: http://www.nnpdf.org/Newsline2015.html#Parker 

Our thoughts and sympathies are with little Parker's family during this time of heartbreak and loss. Please know that your NPD family and community grieves with you and will be keeping all who loved Parker in our hearts in the days, weeks and months ahead. 

Should you need us, know we are here with open arms ~ 

Funding Opportunity for NPC Research ~ Infiniti Coaches Charity Challenge

Dear NNPDF Families and Friends,

The entire Niemann-Pick Disease type C community has been offered an unparalleled opportunity to raise $100,000 towards NPC research, but it will only happen if YOU take action and encourage others to do the same. It's easy! Vote once a day online between now and March 15th to push the Trent and Julie Smith Family Foundation "BReaK Thru Fund" for NPC research to the finish line and the top prize of $100,000! (Three of the Smith family children have been diagnosed with Niemann-Pick Disease type C ~ Braden, Riley and Keaton ~ thus BReaK-Thru Fund.)

Matt Painter, Purdue University Coach and the Smith Family BReaK Thru Fund are working together with the NPC community to vote Matt Painter into the final round! With your help, NPC research WILL garner $100,000, but only if you vote daily! We CAN do this if everyone gets behind this project. 

The first round of voting opened on January 5th, 2015 and continues through January 25th, 2015 when 24 Coaches out of the initial 48 will advance to round two of four total.  Vote daily for Matt Painter to ensure he makes it to the final round!

Share this with your families, share this with your friends and encourage them to do the same.

Alone we are rare, but together we are strong!

To vote, visit: www.votemattpainter.com

For more information about the contest, visit the NNPDF Newsline web page: http://www.nnpdf.org/Newsline2015.html#Infiniti

A Sincere Thank You From the APMRF, DART, SOAR & NNPDF!

Dear NNPDF Families and Friends,

We would like to share this thank you letter, written collaboratively by the Ara Parseghian Medical Research Foundation (APMRF), Dana's Angels Research Trust (DART), Support of Accelerated Research for NPC Disease (SOAR) & the National Niemann-Pick Disease Foundation (NNPDF), with regards to the earlier press release from Vtesse, Inc. 

That announcement dated, January, 7th 2015, highlighted the transition of the National Institutes of Health (NIH) / National Center for Advancing Translational Sciences (NCATS) Cyclodextrin clinical trials from a public project to a public/private partnership with Vtesse, Inc. 

We would like to encourage anyone who wishes to express their sentiments pertaining to this milestone in rare disease research and treatments to do so in the comment section below and we will pass these along to the key stakeholders.

To view the Thank You outside of the Yahoo Group, please visit the NNPDF Newsline website page at: http://www.nnpdf.org/Newsline2015.html#Thanks

2015 NNPDF January e-Newsletter

Dear NNPDF Families and Friends,

The NNPDF January 2015 e-Newsletter is available! Highlights include:

- Clinical Trial News and Updates 

- Infiniti Coaches Charity Challenge 

- 2015 NNPDF Board Meeting 

Click to view the NNPDF January 2015 e-Newsletter: http://www.graphicmail.com/new/viewnewsletter2.aspx?SiteID=4117&SID=0&NewsletterID=1271735

Orphazyme Clinical Trial Updates ~ Press Releases

Dear NNPDF Families and Friends, 

RE: Orphazyme ~ "Orphazyme ApS (www.orphazyme.com) was founded in June 2009 with the aim of developing heat shock protein based therapy for the treatment of diseases caused by defects in the function and/or metabolism of proteins."

The NNPDF is making press release announcements from Orphazyme, pertaining to their advances towards a clinical trial for Niemann-Pick Disease type C, available to the wider NPC Community. For a full archive of Orphazyme updates, visit the NNPDF Orphazyme web page.

Visit this link to the Orphazyme APS (LLC) website to read the press releases: http://www.orphazyme.com/index.php/news/news-releases

“The National Niemann-Pick Disease Foundation (NNPDF) does not engage in the practice of medicine. It is not a medical authority nor does it claim to have medical knowledge. This site is an educational service of the National Niemann-Pick Disease Foundation and is not meant to provide diagnostic or treatment advice. Information contained or suggested on this Web site does not constitute medical advice. For all information related to care, medication or treatment, the NNPDF recommends consulting a physician to determine if information presented is applicable. Please review these additional cautions about medical information provided on the Internet."

Cyclodextrin Clinical Trial Update ~ 01/07/2015

Dear NNPDF Families and Friends,

We are pleased to share with the foundations Niemann-Pick Disease type C (NPC) families and community a recent development pertaining to the Cyclodextrin clinical trial. The recently incorporated, Vtesse, a rare disease company spun-off from Cydan Development, Inc., which is focused on developing drugs for Niemann-Pick Disease Type C (NPC) and other severe diseases with great unmet need, will begin collaborating with the National Institutes of Health on furthering development of Cyclodextrin (VTS-0270) for Niemann-Pick type C.

Vtesse also announced that it has established a Cooperative Research and Development Agreement (CRADA) with the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) and the National Center for Advancing Translational Sciences (NCATS), each a component of the National Institutes of Health (NIH). Vtesse and NCATS have also entered into a licensing agreement for the current rights held by NIH for the worldwide use of cyclodextrin, delta-tocopherol, and derivatives of tocopherol, alone or in combination, for the treatment of lysosomal storage diseases (LSDs), including NPC. Regulatory orphan designations for the U.S. and EU will be also be transferred to Vtesse.

For more information on phase 2 & 3 of the Cyclodextrin clinical trials & the transition from NIH to Vtesse, Inc, please review the full press release here: http://www.nnpdf.org/Cyclodextrin.html#Vtesse

UPDATE: 12:34pm ~ We've added additional links to articles covering the same press release from the National Institutes of Health and the Wall Street Journal (WSJ paid online subscription required) at the above link.

Elisha Elaine Kudrna 04/19/1994 ~ 11/12/2014

Dear NNPDF Families and Friends,

It is with heavy hearts that the NNPDF central office notify the foundation membership of Elisha Elaine Kudrna's passing. Daughter of Jeff & Monica Kudrna of American Canyon, California & beloved sister to Daniel. Elisha lost her battle with Niemann-Pick type A/B on November 12th, 2014. She passed away at home, surrounded by her family at the age of 20.

Elisha's mother, Monica, wrote to us in memory of her beautiful daughter, as well as, the poem included in the attached image:

"She was a bright star in our family, and now she shines eternally in heaven.

She endured so many obstacles in her life, but none of them did she not conquer without a smile. With our faith in God, we know that Elisha had a full purpose in this life, and she is now in heaven with a healed body, dancing and singing loudly. As we miss her with aching hearts every day, we are reassured that we will see her again." 

Our loving support and hearts go out to the Kudrna family as they move ahead without their beloved daughter and sister at their side. Please know that your NPD family and community grieves with you ~ and ~ will be keeping all who loved Elisha and held her dear, in our thoughts and hearts in the days, weeks and months ahead.