Family Support & Medical Conferences
32nd Annual
NNPDF Family Support & Medical Conference
July 11 – 13, 2024
Marriott City Center | Salt Lake City, Utah
Thank you for attending the 2024 NNPDF Family Support & Medical Conference! We hope you found the weekend meaningful and impactful, with new connections made.
We would like to extend a sincere THANK YOU to all of our volunteers, sponsors, and community partners for making our conference a wonderful event for our Niemann-Pick families and community members! It was fantastic to see many of you in support of one another. If you have any questions or comments you’d like to share with us, please contact us at [email protected].
2024 Conference Recordings
Community Impact Updates
- NNPDF & INPDA Updates
- Keynote Address: Dr. Klaus Romero
- Niemann-Pick Diseases: An overview of the diseases for families
- Digital Health Technologies Update
- Tips & Tactics for Living with Niemann-Pick Disease: IEPs
- Newborn Screening
- Research: International Niemann-Pick Disease Registry (INPDR)
- Super Siblings Panel Discussion
- Experts Panel Discussion: Continuing the Advancement of Diagnosis and Treatment of Niemann-Pick
ASMD Updates
NPC Updates
Awards
2024 NNPDF Persevere Award
Congratulations to Darrile Papier our 2024 Persevere Award recipient. Read more.
2024 NNPDF Cora Sterling Endurance Award
Congratulations to Charlotte Joy Norris our 2024 NNPDF Cora Sterling Endurance Award recipient! Read more.
Thank You to our Family Support & Medical Conference Sponsors
NNPDF greatly appreciates the efforts of our industry partners in helping to find treatments and support on behalf of our Niemann-Pick community. THANK YOU for your generous support of our 32nd Annual Family Support & Medical Conference.
~ SAVE THE DATE ~
NNPDF Family Support & Medical Conference
July 10 – 13, 2025
Embassy Suites by Hilton Charlotte/Concord Golf Resort & Spa | Charlotte, NC
31st Annual
NNPDF Family Support & Medical Conference
July 20 – 22, 2023
Wyndham Grand | 14651 Chelonia Parkway | Orlando, Florida
THANK YOU to all that joined us in-person and virtually for our 31st Annual Family Support & Medical Conference! We were so happy to see all those who joined us! It was a wonderful time of connecting with one another, sharing, learning, and supporting each other.
Our Family Support & Medical Conference was a success due to many supporters: from our sponsors and speakers, to our board and staff, and of course our fantastic volunteers! We THANK YOU for all that you have done to support our family conference.
2023 Conference Recordings
Community Impact Updates
- Advocacy Hot Topics - Round table discussion with our experts
- Clinical Update: Adrabetadex
- Keynote Address: Overcoming Obstacles in Rare Disease
- NNPDF Research Fellowship Updates
- Research: Efavirenz
- Research: Gene Therapy
- Research: International Niemann-Pick Disease Registry (INPDR)
- Siblings Panel Discussion
ASMD Updates
NPC Updates
Awards
2023 NNPDF Breakthrough Award
Congratulations to Sanofi our 2023 NNPDF Breakthrough Award recipient! Read more.
2023 NNPDF Cora Sterling Endurance Award
Congratulations to Liam Ferguson our 2023 NNPDF Cora Sterling Endurance Award recipient! Read more.
2023 NNPDF Persevere Award
Congratulations to SOAR (Support of Accelerated Research) our 2023 Persevere Award recipient. Read more.
Thank You to our Family Support & Medical Conference Sponsors
NNPDF greatly appreciates the efforts of our industry partners in helping to find treatments and support on behalf of our Niemann-Pick community. THANK YOU for your generous support of our 31st Annual Family Support & Medical Conference.
~ SAVE THE DATE ~
NNPDF Family Support & Medical Conference
Salt Lake City, Utah | July 11 – 13, 2024
30th Annual
NNPDF Family Support & Medical Conference
July 28 – 31, 2022
Wyndham Grand | 14651 Chelonia Parkway | Orlando, Florida
THANK YOU to all that joined us in-person and virtually for our 30th Annual Family Support & Medical Conference! We were so happy to see all your smiling faces . It was a wonderful time of sharing, supporting and learning.
Our Family Support & Medical Conference was a success due to many supporters: from our sponsors and speakers, to our board, staff and volunteers. We THANK YOU for all that you have done to support our family conference.
2022 Conference Recordings
Community Impact Updates
- Advocacy Round Table: Unmet Needs in NPC & ASMD
- International Niemann-Pick Disease Registry (INPDR)
- Keynote Address and Remarks: Challenges and Opportunities in Rare Disease
- Medicaid Waivers and Navigating Insurance Benefits
- Newborn Screening
- NNPDF Advocacy Update
- Research: Advances in Gene Therapy
- Research: International Niemann-Pick Disease Registry (INPDR)
- Research: Update on Strategies to Enhance the Endocannabinoid System in ASMD and NPC
ASMD Updates
NPC Updates
Awards
2022 NNPDF Persevere Award
Congratulations to Dr. Ed Schuchman our 2022 Persevere Award recipient. Read more.
2022 NNPDF Cora Sterling Endurance Award
Congratulations to Christopher Sousa our 2022 NNPDF Cora Sterling Endurance Award Recipient! Read more.
2022 Joele Ruppert & Joseph Colton ASMD Scholarship
Congratulations to Yasmin Markman our 2022 Joele Ruppert & Joseph Colton ASMD Scholarship recipient.
Congratulations to Jack Visoky our 2022 Joele Ruppert & Joseph Colton ASMD Scholarship recipient.
Thank You to our Family Support & Medical Conference Sponsors
NNPDF greatly appreciates the efforts of our industry partners in helping to find treatments on behalf of the Niemann-Pick community. THANK YOU for your generous support of our 30th Annual Family Support & Medical Conference.
29th Annual
NNPDF Family Support & Medical Conference
July 29 – August 1, 2021 | Virtual Conference
THANK YOU to all that joined us for our 29th Annual Family Support & Medical Conference! We were so glad to have you join us virtually! It was a wonderful time of sharing, supporting and learning.
Our Family Support & Medical Conference was a success due to many supporters: from our sponsors and speakers, to our board, staff and volunteers. We THANK YOU for all that you have done to support our family conference.
2021 Conference Recordings
Awards
2021 NNPDF Persevere Award
Jim & Missy Ward
Congratulations to Jim & Missy Ward, 2021 Persevere Award recipients! Read more.
2021 NNPDF Cora Sterling Endurance Award
Alec Koujaian
Congratulations to Alec Koujaian, our 2021 NNPDF Cora Sterling Endurance Award Recipient! Read more.
ASMD Updates
Community Impact Updates
- Keynote Address: Power of Patient Advocacy
- Value of Patient Owned Data
- Advocacy: Amplifying Our Voices in the Rare Disease Community
- NNPDF Family Services
- NNPDF Collaboration: Young Adults, Young Leaders
- How to Communicate Your Message to the Media Breakout Session
- Medicaid Waivers Breakout Session
- Newborn Screening: Roadmap & Updates
- NNPDF Research Fellowship Updates
- Research: Endocannabinoids
- Biomarkers as Surrogate Endpoint & Their Importance in Clinical Development
- Interactive Roundtable Discussion: Challenges facing the Niemann-Pick Disease Community
Family Journeys
NPC Updates
NNPDF greatly appreciates the efforts of our industry partners in helping to find treatments on behalf of the Niemann-Pick community. THANK YOU for your generous support of our 29th Annual Family Support & Medical Conference.
28th Annual
NNPDF Family Support & Medical Conference
July 10 – 12, 2020 | Virtual Conference
THANK YOU to all that joined us for our 28th Annual Family Support & Medical Conference! We were so glad to have you join us virtually! It was a wonderful time of sharing, supporting and learning.
Our Family Support & Medical Conference was a success due to many supporters: from our sponsors and speakers, to our board, staff and volunteers. We THANK YOU for all that you have done to support our family conference.
2020 Conference Recordings
Awards
2020 NNPDF Cora Sterling Endurance Award
Molly Honsinger & JP Honsinger
Congratulations to Molly & JP, our 2021 NNPDF Cora Sterling Endurance Award recipients! Read more.
2020 NNPDF Persevere Award
Lisa Chavez
Congratulations to Lisa Chavez, 2020 Persevere Award recipient! Read more.
ASMD Updates
Community Impact Updates
- Keynote Address: The Impact of COVID19 on the Regulatory Process for Rare Diseases
- COVID19 Impacts & Effects on the Niemann-Pick Community: Panel Discussion with Treating Physicians
- International Niemann-Pick Disease Registry
- Newborn Screening and the “ScreenPlus” Program Update
- Understanding the American Niemann-Pick Community: Insurance Survey Update
Family Journeys
NPC Updates
NNPDF greatly appreciates the efforts of our industry partners in helping to find treatments on behalf of the Niemann-Pick community. THANK YOU for your generous support of our 28th Annual Family Support & Medical Conference.
27th Annual
NNPDF Family Support & Medical Conference
August 15 – 18, 2019 | Bloomington, Minnestota
Hilton Minneapolis/St. Paul Airport
3800 American Boulevard East | Bloomington, MN 55425
THANK YOU to all that joined us in Bloomington for our 27th Annual Family Support & Medical Conference! We were so glad to have you there! It was a wonderful time of fellowship, sharing, supporting and learning.
2019 Persevere Award and Endurance Award Recipients!
2019 NNPDF Persevere Award
Dr. Melissa Wasserstein
Congratulations to Dr. Melissa Wasserstein, 2019 Persevere Award recipient! Read more.
2019 NNPDF Cora Sterling Endurance Award
Serina Heinze
Congratulations to Serina Heinze, our first annual NNPDF Cora Sterling Endurance Award recipient! Read more.
Our Family Support & Medical Conference was a success due to many supporters: from our sponsors and speakers, to our board, staff and volunteers. We THANK YOU for all that you have done to support our family conference.
NNPDF greatly appreciates the efforts of our industry partners in helping to find treatments on behalf of the Niemann-Pick community. THANK YOU for your generous support of our 27th Annual Family Support & Medical Conference.