August 2018 Newsletter
Message from the Executive Director
Dear Friends and Families,
This Newsletter comes to us as summer is winding down, many children are returning to school and we are readying for the fall. It was such a delight to meet so many of you earlier this month in Kentucky at this year’s Family Conference. It was amazing to see families come together with the ability to celebrate milestones with some, while still sharing the pain of loss with others.
It’s clear that we are a special community and have so much to share with one another. NNPDF was honored to provide this special opportunity for families to connect. We greatly appreciate those who helped financially sponsor the event and very special thank you to all of you who volunteered in so many ways over to make the conference the success it was.
The weekend was full of insightful presentations from all our industry partners, with encouraging updates on current research that is continuing to progress us to a cure. There were many break out groups where families where able to deepen their relationships through commonalities and support, while also educating us on what is important to you moving forward. As with every year, Saturday Night’s Gala was the hit of the conference. Seeing the joy on so many faces as the Activity Zone performed for us and we danced the night away was the perfect ending to our time together. You can see pictures from the Gala night and throughout the conference here.
Many of you met Laurie Turner, who we introduced at the conference as our new Family Service Manager for our pilot program. Laurie brings 15 years of experience with family services programs at the MPS Society and has already begun connecting with the community members. Laurie can be reached here and is available for consultations, questions, information referrals, or just a supportive call.
This continues to be an exciting time of hope for us as a community. We are fortunate to have multiple clinical trials ongoing, along with many research programs with regards to all types of Niemann-Pick Disease. The landscape of this rare disease will continue to evolve and change in the coming years. As the national patient organization, we will continue to remain at the forefront and to share all available information with you in an ethical and unbiased manner, while advocating on behalf of the community.
I hope that you enjoy these final weeks of summer.
Joslyn Crowe, MSW, MA
NNPDF Executive Director
New Resource Available to NPC Community
The NNPDF has developed a patient handbook (in a downloadable PDF) for NPC patients and families titled My Health Handbook & Emergency Information. This booklet provides NPC patients a place to record important and detailed medical information to share with medical personnel in case of an emergency. To download the handbook click here.
Clinical Trial Updates
UPDATE: Mallinckrodt Announces an Open-label Expanded Access Treatment Protocol for VTS-270. 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.
NNPDF 26th Annual Family Conference
The NNPDF 26th Annual Family Conference was held from August 2 -5 in Louisville, Kentucky. THANK YOU to all of the families, speakers, and representatives that joined us this year. It was a time of educating, sharing and fun! Each of you that attended played a part in making the event outstanding and truly special.
This year’s Conference was highlighted by sessions on Clinical Trials, Drug Development, Research Updates, Dr. Marc Patterson receiving the Persevere Award (click here for video), specialist-facilitated grief support sessions, disease support breakout sessions, the International Niemann-Pick Disease Patient Registry, Family Services and Advocacy, and a Meet & Greet with Pharma. An extra special thank you to our Activity Zone leaders and volunteers who created an energetic and caring atmosphere for the children, young adults, and siblings, complete with a performance at the Gala dinner!
Family Services Update
Hello! I am so honored to be here as the new Family Services Manager at the NNPDF. It was wonderful to attend the Annual Family Conference which allowed the opportunity to meet so many of you – thank you for sharing your family and stories with me. Over the next couple of weeks, I will be reaching out to our US based members. I look forward to connecting with you and learning from your experiences. Please do not hesitate to reach out to me first – you can reach me via email at email@example.com or telephone 920.563.0930.
NNPDF Family Services Director
We are seeking volunteers!
NNPDF is collecting resumes and letter of interest from those interested in volunteering skills and time to assist with focus groups, committee teams and even as a Board member. If you are interested in becoming a NNPDF volunteer please let us know at firstname.lastname@example.org and we will follow up with you with the process for applying.
Community Industry Feedback
Pharmaceutical companies are limited in how much they can interact with patients/families they therefore rely on a national patient group, like the NNPDF, and work with us to understand issues that are important to our community. As a reminder, we have developed a community communication system so that NNPDF would have a way to give feedback in a collective manner. This mechanism for coalescing family and patient feedback is fully supported by all of the pharmaceutical companies currently engaged in the trials. If you haven’t already, we invite you to provide feedback relevant to our industry partners. To learn more about this program, please follow the link below.
Missy Ward, NNPDF Board Secretary, has initially volunteered to take your email feedback with the header “Community Industry Feedback”. You can reach Missy at email@example.com. You may also send her your phone number if you are more comfortable providing feedback over the phone. Please allow 1-3 business days for response.
NNPDF Equipment Exchange
Pre-owned equipment can be a huge help to others in our NPD community. Through the NNPDF Equipment Exchange program, the foundation facilitates any equipment requests and donations of pre-owned equipment to help others in our community who may be in need. Click here for more equipment exchange program information. If you have any questions or an interest in any items, please contact the NNPDF at firstname.lastname@example.org.
Ramps and Cargo Carriers
The NNPDF was recently contacted by a local business of mobility products. They have generously offered to donate some of their items to help families affected by Niemann-Pick Disease which include threshold ramps, wheelchair ramps, and tray cargo carriers, subject to availability. Thank you to Discount Ramps for your generosity.
For information on items offered by Discount Ramps and other available items click here.
We asked our families for tips and tricks to help make life a bit easier and you all delivered! Here are a few helpful tips…
TIP #1: SimplyThick is a wonderful thickener that helps with swallowing of liquids that comes in a gel and mixes easily. Available in a large bottle with a pump for your counter or convenient travel packets that come in various thicknesses. Visit simplythick.com for information.
TIP #2: Baby leg warmers can help keep legs warm instead of pants. They minimize discomfort of diaper changing when it is cold. Leg warmers also work well on the arms if they are cold in place of long sleeves.
TIP #3: To help prevent your loved one from sliding down their mattress, sew a cloth diaper to a fitted sheet.
If you have helpful tips or tricks please send them to email@example.com.
Shop and Support the NNPDF
If you enjoy shopping online, you can do so AND support the NNPDF! How? Simply click here for information and for registration. Remember to choose the National Niemann-Pick Disease Foundation as your supporting cause. With every purchase these supporters donate a portion of your purchase to the NNPDF. You shop… They give. Don’t forget to share these links with your friends!
Fundraising and Awareness Opportunities
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.
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 firstname.lastname@example.org to let us know.
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.