May 2024 Newsletter

Message from our Executive Director

Dear Friends,

We are only 6 weeks away from our annual Family Support & Medical Conference! I am so excited to see everyone in Salt Lake City this July. The conference is such a highlight in our year – a weekend of meaningful connections, new friendships, education, scientific updates, and most of all, of belonging.

I am thrilled to announce our keynote speaker as Dr. Klaus Romero from Critical Path Institute (CPath). Dr. Romero will share an update on the fascinating and important accomplishments of the Critical Path for Lysosomal Disease Consortium. NNPDF is an active member of this consortium which unites regulators, pharmaceutical and biotech companies, academics, and patient advocacy organizations to advance drug development in diseases including Niemann-Pick.

In addition to our first-rate educational sessions, family services sessions, and incredibly FUN gala dinner, there is plenty of time for gathering and connecting with other Niemann-Pick families. Don’t miss the chance to purchase discounted tickets for a great night of baseball as the Salt Lake Bees take on the River Cats at Smith’s Ballpark on Friday, July 12th at 6:35pm.

Some important conference deadlines are approaching – see below for all the details!

I look forward to seeing you in Salt Lake City!

Kind regards,

Joslyn Crowe
NNPDF Executive Director

July 11-13, 2024 | Salt Lake City, Utah

Be sure to watch NNPDF communications for important updates. Visit nnpdfconf.org for all conference information including Things to Do in the Salt Lake City area!

Conference Registration

Conference Shirt Deadline – Register by June 10th to ensure your complimentary 2024 NNPDF Family Support & Medical Conference shirt will be waiting for you at the conference!

First Time Attendee Discount: Enjoy a 10% discount off your registration! We’re happy to have you join us!

June 28th is the final day for registration so please be sure to complete your registration by this important deadline.

Accommodations

Reserve by June 24th

NNPDF group rate will be available until June 24th, 2024. NNPDF Group Rate is $159 ($184.15 including taxes) per night. The NNPDF hotel group rate is available three days before and three days after the conference, dependent on hotel availability.

Salt Lake Bees Baseball Game

Special Promotion for NNPDF Family Conference Attendees

Join other NNPDF families for a great night of baseball as the Salt Lake Bees take on the River Cats at Smith’s Ballpark on Friday, July 12 at 6:35 pm. (Please note that you will need to make your own transportation arrangements as it will not be provided.) Ballpark information can be found at milb.com/salt-lake/ballpark

Get your NNPDF Group tickets at t.ly/SaltLakeBees-NNPDF

Speaker Announcements

Special Offer for Conference Attendees

We are excited to share a special offer for Family Support and Medical Conference attendees.

Order your NNPDF gear by June 11th and take advantage of FREE SHIPPING when you pick up your merchandise at the NNPDF Family Support & Medical Conference!

It’s easy!

Select your gear and place your order
In Shipping Method select “PICK UP AT CONFERENCE for FREE”
Grab your gear at the conference!

I Need A Cure for NPD - ASMD - Store Image

Someone I Love Needs a Cure - NPC - Store Image

NNPDF Upcoming Events

NNPDF and Niemann-Pick Canada present Update from the International Niemann-Pick Disease Registry
May 31, 2024 | 1:00pm ET
Register in advance.

NNPDF In Action

NNPDF’s Executive Director, Joslyn Crowe, and Family Services Manager, Laurie Turner led a roundtable discussion at World Orphan Drug Congress USA focused on The Impact of Rare Disease on Siblings. NNPDF Board Members Taylor Sabky (center) and Paul Merrigan (not pictured) also represented NNPDF at the conference.

Justin Hopkin, NNPDF Scientific Advisory Board member, was a featured speaker at the EveryLife Foundation Scientific Workshop. Justin discussed “Challenges in Very Small Population Therapy Development” as related to Niemann-Pick type C. Joslyn Crowe, NNPDF Executive Director, was in attendance as a member of the workshop’s planning committee.

Milestones

Congratulations to Evren Ayik on his recent graduation from California State University Fresno. Evren earned his bachelor’s degree in Liberal Studies and graduated with Magna Cum Laude honors. He will be returning in the fall to earn his teaching credentials and master’s degree. Way to go Evren!

Congratulations to Morgan Hill! Morgan has achieved a significant milestone as part of the 2024 graduating senior class at Syracuse High School. Despite the challenges she faced, Morgan persevered, stayed strong, and successfully completed her high school journey. Way to go Morgan!

If you have a special Milestone you’d like to share with your Niemann-Pick community please send us a photo and the details at nnpdf@nnpdf.org.

Fundraising

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.

THANK YOU to Laura Fischer, Brittany Gittings, David Nead, Jessica Martin, and Macenzie Susan Waits who recently held a fundraiser to help support NNPDF’s mission!

If you have recently hosted a fundraising event, send us your photos and we’ll share them and details from your event in upcoming newsletters! Want to host your own fundraiser? Facebook Fundraisers are an easy option! Visit facebook.com/fund/NNPDF to get started!

Comprehensive Care Centers

NNPDF provides a listing of medical institutions that have experience in caring for and treating Niemann-Pick disorders. This project is supported by our Scientific Advisory Board and we are proud to offer this valuable resource to our families as they search for medical care and treatment. This information has been provided to us by the institutions. Click here to learn more!

If you are connected with a medical team that would like to be added to the NNPDF Comprehensive Care Centers listing, please contact Laurie Turner at lturner@nnpdf.org.

Family Assistance & Support Program

The NNPDF Family Assistance & Support Program (FASP) provides relief to NNPDF U.S. member families that may be facing a financial obstacle that requires short-term financial assistance. Funding includes but is not limited to, specialized medical equipment and goods, utility bills, adaptive home repairs, home and car repairs, car payments, rent and mortgage payments, and bereavement expenses. Learn more about the NNPDF Family Assistance & Support Program.

Please contact Laurie Turner, Family Services Manager at 920-542-4038 or familyservices@nnpdf.org if you have any questions about this program.

ASMD Accelerate

Clinical Trial Updates

Clinical trials are currently in progress to study and develop treatments for ASMD and NPC. The NNPDF posts new information regarding clinical trial updates as soon as it is received. Visit our Clinical Trials web page for up to date information on all clinical trials.

The Assistance Fund

Struggling with out-of-pocket expenses due to acid sphingomyelinase deficiency (ASMD)? The Assistance Fund is an independent charitable patient assistance organization helping patients and families by providing financial assistance for the cost of treatment and other health-related services. Through its Acid Sphingomyelinase Deficiency Financial Assistance Program, eligible individuals can receive assistance for all FDA-approved treatment for ASMD. Learn more.

NNPDF Membership

Enrolling, confirming, or updating your membership will ensure we have accurate information for you and your family. This will help us to continue to provide you with important notifications and updates from the NNPDF.

Click here to update or enroll today!

For assistance contact Laurie at familyservices@nnpdf.org or call 920-542-4038