NNPDF ASMD News: FDA Grants Breakthrough Therapy Designation for Genzyme’s Olipudase Alfa

Dear NNPDF Families and Friends,

The NNPDF Central Office is pleased to share a Press Release from Genzyme, a Sanofi Company, (dtd:  Thursday, June 4th, 2015) which announces that the United States Food and Drug Administration (FDA) has granted “Breakthrough Therapy” designation to olipudase alfa. This enzyme replacement therapy (ERT) is being investigated for the treatment of patients with nonneurological manifestations of acid sphingomyelinase deficiency (ASMD), also known as Niemann-Pick Disease type B. 

Breakthrough Therapy designation is intended to expedite the development and review of investigational new drugs that target serious or life-threatening conditions which have an unmet therapy or medical treatment. 

Please visit the NNPDF Newsline page (http://www.nnpdf.org/Newsline2015.html#Jun4th2015) for access to the full press release, as well as, to learn more information about the FDA “Breakthrough Therapy” designation and the impact that this FDA designation will have on the upcoming pediatric and adult clinical trials in the United States.

Support These NPD Awareness Events This Weekend!

Dear NNPDF Families and Friends,

The NNPDF Central Office would like to remind our family membership that there are two NPD related events taking place this weekend!

The first event is being hosted by the Phil and Andrea Marella Family in loving support of their children with NPC, Dana and Andrew.

Gladys Knight in Concert for DART

Saturday, May 30th, 2015

Palace Theatre, Stamford, CT

On Saturday, May 30th, 2015, Gladys Knight will perform for Dana's Angels Research Trust (DART)!  7-time Grammy Winner GLADYS KNIGHT will headline DART's 2015 Gala Concert for NPC research! Come ride THE MIDNIGHT TRAIN... to Stamford!

The concert will kick-off with a Gala Benefit that includes an elegant cocktail reception with a pasta bar by World-Famous RAO’s Italian Restaurant, silent and live auctions, and celebrity hosts Kathie Lee and Frank Gifford. Scott Shannon of WCBS-FM 101.1 will introduce Gladys Knight!

For more information on the event and to purchase tickets you can go to http://danasangels.org/gladys-knight-in-concert-for-dart/

The second event is an event in support of the International Niemann-Pick Disease Alliance (INPDA). 

Felix Schleuniger from Switzerland will start his 2,500 mile Great Mountain Bike Ride on Sunday, May 31st, 2015. 

Please join us in Albuquerque on May 31st to support Felix and Niemann-Pick patients worldwide. 

Felix is riding and fundraising for the International Niemann-Pick Disease Alliance, a support organization for those affected by Niemann-Pick Disease. 

Lisa Chavez, Co-Vice Chair of the NNPDF, her family and other Niemann-Pick families from the nearby community of Albuquerque, New Mexico will be meeting Felix at the start-line of his trek and wishing him good luck on his arduous journey.  

Please share this information and the INPDA FaceBook page developed in support of Felix with all of your friends and be certain to follow Felix along on his adventure!  http://www.facebook.com/inpda.gdmbr

Keaton J. Smith (NPC) 09/24/2000 ~ 05/22/2015

Dear NNPDF Families and Friends,

It is with a heavy heart that we notify our NPD family membership of the passing of Keaton J. Smith (September 24, 2000 - May 22, 2015 ~ NPC), who courageously fought the NPC battle for 14 years. Keaton played on the Tippy Stars Baseball Team and was a huge fan of Purdue men’s and women’s basketball. He was an 8th grader at Battle Ground Middle School and a member of St. Thomas Aquinas Center in West Lafayette, Indiana. 

Keaton’s family noted that they draw some comfort in knowing that he is now playing with his brother Braden and sister Riley who both also suffered from Niemann-Pick Disease and passed away in 2006 and 2014 respectively.

To Keaton’s loving parents, Trent and Julie, and beloved older sister, Chandlar, 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.

Keaton’s obituary can be found at http://www.soller-baker.com/memsol.cgi?user_id=1588635.

May 2015 e-Newsletter ~ Now Online

Dear NNPDF Families and Friends,

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

• 23rd Annual NNPDF Family Support and Medical Conference
• Update ~ A Message from your Foundation Board of Directors! 
• NNPDF Membership ~ Action Required 
• NNPDF Fellowship Research Progress Reports
• Clinical Trial News & Updates 
• Fundraising & Recipes for Success 

Click below to view the NNPDF May 2015 e-Newsletter: http://www.graphicmail.com/new/viewnewsletter2.aspx?SiteID=4117&SID=0&NewsletterID=1303883

Orphazyme Clinical Trial Development Update on Arimoclomol in NPC

Dear NNPDF Families and Friends,

The National Niemann-Pick Disease Foundation has been advised by Orphazyme that an “Observational” clinical study for NP-C, tied to their clinical development program of arimoclomol in NPC, has been registered and made available on www.clinicaltrials.gov. The link to the information pertaining to just this trial is at: https://clinicaltrials.gov/ct2/show/NCT02435030?term=02435030&rank=1. 

This information was updated as of April 30, 2015. 

At this time, no site in the United States has been announced and currently the study is not open for participant recruitment. Orphazyme has indicated that they are targeting a clinical trial to start date in the United States toward the end of first quarter 2016. As we receive further announcements and updates, it will be posted to our NPD family community! 

You can read either the full text view or the tabular view depending upon your preference. The text allows you to see the inclusion and exclusion information along with the primary outcome measures they will be looking at. 

Sincerely,

Nadine M. Hill

Executive Director

National Niemann-Pick Disease Foundation

Happy Mother's Day from the NNPDF!

To all our NPD Families and Friends,

Many of our NPD Mom’s will be joining will family in celebration of Mother’s Day around the world this Sunday. May your day, and all those to follow, be filled with love and joy. 

~ NNPDF

A Re-Introduction to the Canadian Chapter of the National Niemann-Pick Disease Foundation

Dear NNPDF Families and Friends,

Many of you may not be aware of the fact that NNPDF has a sister chapter in Canada. The Canadian Chapter of the National Niemann-Pick Disease Foundation (CCNNPDF) and the NNPDF work collaboratively to help support families and research to find treatments and a cure for all types of Niemann-Pick Disease. The NNPDF office in the United States provides administrative, marketing and family support for the CCNNPDF. Please see the new CCNNPDF e-newsletter to learn more about what is on the horizon for our families and friends in Canada.

Click here to view the CCNNPDF e-Newsletter.

All my best, 

Nadine Hill

NNPDF Executive Director