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January 2019 Newsletter

JANUARY 2019


Message from the Executive Director  |  Family Services Update
Build With A Bear  |  Clinical Trials Update
PALS – Patient AirLift Services  |  Rare Disease Day 2019
Upcoming Events  |  NNPDF Equipment Exchange
Fundraising  |  Stay Connected With Us


Message from the Executive Director

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Dear Friends and Families,

The new year at NNPDF is off to an energetic start!  There is always a feeling amongst our dedicated staff and leadership team of “How can we best serve the community? How can we help more? What else is needed?” and we appreciate the feedback from you that we have received so far.  We are busy planning more community support initiatives and assistance programs which will be announced in the coming months.

Today, we are thrilled to launch our new logo, and with it our updated website and Newsletter! We’ve come a long way over the past few years, as a community and as an organization. Our logo has evolved has over the past 26 years to reflect our Foundation’s history and various stages of development. We began with a rainbow based on a drawing created by a child with Niemann-Pick Disease which symbolized hope. Over time as our organization grew, our Board worked to keep the spirit and inspiration of this original drawing as an underlying promise of hope. Our new logo reflects the next phase of the National Niemann Pick Disease Foundation’s growth. This new image was designed to reflect the positive spirit of the community, to continue the hopeful nature of the rainbow’s arc, and to capture the essence of those who have passed with the butterfly image.

Over the past 26 years, we have grown from a few collaborating families to the national patient organization for the Niemann-Pick disease community, representing families from throughout the US in all stages of their Niemann-Pick journey, with special focus on advocacy, family supports and services, and research. We are so grateful to each family who has been a part of developing and sustaining our organization over the past 26 years through your combined volunteer efforts, resources and knowledge. 

Throughout 2019 NNPDF will actively support the community through our continued emphasis on advocacy, which is central to our core values and our mission.  We believe it is important to be a champion for the community and to be a driving force of positive change.  We will continue to advocate on issues related to and impacting the Niemann-Pick community at the federal, state, and regional levels.

We will continue to memorialize those who have passed while pursuing hope for future children with NPD through our continued discussions with the FDA for the NPC and ASMD communities, and unbiased support of industry to help advance clinical trials to become regulatory-approved treatment options. We will also continue to collaborate in community efforts that support progressive movement through our efforts to educate the community and facilitate enrollment in the International Niemann-Pick Disease Registry, our support of the PFDD meeting taking place in March and our active support of newborn screenings initiatives that may make early detection and diagnosis possible for future families.

We will expand our webinar series and include an Industry Update series bringing you the latest news and updates from the biotech and pharmaceutical companies working on treatments and cures for Niemann-Pick disease. Our family services program, that was clearly defined a priority by our national community, continues to grow and Laurie, our Family Services Manager, is available to help with your individual needs and concerns.

With so many exciting NPD community initiatives and a fresh new look, 2019 promises to be a year of continued growth for NNPDF and the community we serve. As we work together this through advocacy, family support, and research, we continue to do with respect for all who have supported our journey over the last 26 years and renewed hope for patients and families facing NPD.

Warmly,


Joslyn Crowe, MSW, MA
NNPDF Executive Director

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Family Services Update

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I am delighted to be able to start this new year with each of you. Last August, the NNPDF launched a pilot Family Service Program in response to the growing needs of patients and families, with a conscientious effort to begin meeting those needs, along with delivering on the organization’s mission statement and vision for the future. Due to the overwhelming success of this pilot program and continued generosity of donors and fundraisers, I am pleased to share with you that Family Service Program will remain an active program of the NNPDF, to which I’m honored to lead.  

The past 6 months have been devoted to getting to know you as individuals and also community members. Through outreach and community conversations we were able to begin developing programs that are in direct correlation with needs expressed from families.

One of those programs is Family Connection Chat, which we kicked of last week. This will be a regular event happening 1-2 times each month. Our families indicated they wanted and needed a way to stay connected. Family Connections Chats are video or audio chats, allowing us to talk and share together on the topics you want to discuss. Our next chat will be January 30th and our topic will be “Sharing your Diagnostic Journey”. This is a great opportunity to share your insights and knowledge, as well as learn from others and better understand each others’ individual journeys. Topics will vary each month, please let me know what you would like to discuss with others or if you would like to be included in our next discussion.

I feel fortunate to have met many of you in person at different conferences and workshops. The NNPDF will be supporting the efforts of many NPC families who are gathering in Washington, DC in March for a Patient Focused Drug Development (PFDD) meeting. I will be there and would love to meet with any of you who will be in attendance. The PFDD meeting is a NPC community effort to help the FDA to obtain the patient perspective of NPC patients and their daily lives. Click here if you would like to register to attend this meeting or learn more about it.   

As always, please feel free to reach out to me via phone at 603-413-8707 or email familyservices@nnpdf.org, as I mentioned above, I am working on several projects and your input will be helpful! Thank you for your continued involvement with the NNPDF and our efforts to support all families through their journey. I look forward to continuing to build relationship with each of you throughout 2019. 

Regards,

Laurie J. Turner
Family Services Manager

familyservices@nnpdf.org

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Build With A Bear

We are excited that this week the Hilton Rosemont in Chicago will host its Build With A Bear event in honor of the NNPDF. Several local families will join their team and build unique teddy bears with a former player from the Chicago Bears, to raise awareness of Niemann-Pick disease. Follow the event through our social media Facebook and Twitter pages and look for photos in the February Newsletter.

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Clinical Trial Updates

Updates from CTD Holdings, Inc.:
CTD Holdings to present at upcoming Brains for Brain conference on lysosomal storage diseases. Click here for complete announcement.

CTD Holdings announces partnership with United BioSource LLC to support the extension protocol for its Phase I Clinical Trial in the United States. Click here for complete announcement.

Update from Sanofi Genzyme:
Sanofi Genzyme announces ASCEND and ASCEND-Peds studies complete enrollment. Click here for complete announcement.

The NNPDF posts new information regarding clinical trial updates as soon as it is received. Please review our Clinical Trial Update procedure for alerting you to these updates as they are made. READ MORE

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PALS – Patient AirLift Services

PALS is a non-profit charitable aviation organization that arranges free flights in the greater Northeast region through their volunteer aviation community for individuals requiring medical diagnosis, treatment or follow-up, or for compassionate assistance. READ MORE

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Rare Disease Day 2019

Rare Disease Day 2019 will focus on bridging the gaps in the coordination between medical, social and support services in order to tackle the challenges that people living with a rare disease and their families around the world face every day. Rare Disease Day® takes place annually on the last day of February. Anyone can get involved in Rare Disease Day awareness. Click here to learn how you can participate!

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JANUARY
Family Service Connections Video Chat:
Sharing Your Diagnostic Journey
Wednesday, January 30th | 8:00 pm EST/5:00 pm PST
To register contact Laurie at familyservices@nnpdf.org

“Me, too.” Two words that can help anyone feel a little better in whatever stage they are at in their NPD journey. In this chat you will be given the opportunity to share the journey you and your family encountered in leading up to the diagnosis of Niemann-Pick Disease. Your experience and insight may be helpful to someone else who is just beginning their own NPD journey.

FEBRUARY
Rare Disease Day
February 28, 2019
Click here for details on how to get involved and get your personalized Facebook profile picture!

Industry Updates Webinar: TBA

MARCH
NPC Patient-Focused Drug Development Meeting
Monday, March 18, 2019 | Hyattsville, Maryland
Click here for information.

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NNPDF Equipment Exchange

The NNPDF Equipment Exchange program assists those who wish to donate pre-owned equipment to others who may be in need. Click here to check out all items available. If you have any questions or an interest in any items, please contact the NNPDF at nnpdf@nnpdf.org.

New Items Available:

 

Cough Assist Airway Clearing Device

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Shower Chair

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Fundraising and Awareness Events

Contributions through fundraising by NNPDF members, families and friends are used to provide services and information to individuals and families affected by NPD, as well as supporting research into finding treatments. Please continue to host and support NPD fundraisers. Awareness Events promote awareness to the general public about Niemann-Pick Disease. Click here to view current and past fundraising and awareness events.

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Facebook Fundraisers

THANK YOU to those of you who have already hosted a Facebook Fundraiser! Are you interested in hosting? It is a great opportunity to raise awareness and funds for the NNPDF. If you are hosting a Facebook fundraiser please email the NNPDF Central Office at nnpdf@nnpdf.org to let us know.

Have you hosted a fundraiser for NNPDF recently? Send us your photos and we’ll share them and details from your event in upcoming newsletters!

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Stay Connected With Us

A quick reminder that if you are not receiving our NNPDF monthly newsletter via email or have recently changed your address or email, please click here to join us or to update your information.

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1/22/2019 jjb
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