Surveys, Studies, & Market Research
Surveys, studies, and market research play a critical role in deepening our understanding of the needs and experiences of individuals and families affected by Niemann-Pick disease. Your participation contributes essential data that informs scientific research, guides the development of treatments, enhances support services, and strengthens advocacy efforts across the community.
NPC Caregiver Sentiment Research
NNPDF would like to share the following message and invitation below from Beren Therapeutics with the NPC community to share their perspectives and experiences as caregivers on the treatment and management of their child with NPC.
As a Public Benefit Corporation, Beren is working to ensure that we are aligned with the patient and caregiver NPC community to provide the most valuable and relevant information and materials to support the NPC community. As such, we are conducting a market research project to gather your insights into the lived experience of NPC families regarding their medical care and treatment journey. Specifically, we’d like to hear about:
- Your child’s treatment and disease management experience.
- Your views on potential future therapeutic options and treatment processes.
Study Details:
- Format: Individual 60-minute interview via Zoom
- Scheduling: Interviews will be scheduled at your convenience over the coming weeks.
- Participant Incentive: As a thank you for your participation, each participant will receive $175, which will be paid via check.
Who can participate?
We’re seeking caregivers of children with infantile-onset NPC who have not participated in the Mandos Expanded Access Program (EAP) for adrabetadex.
How to indicate interest: To participate or learn more, contact: Ally Larson at [email protected]
Important Information:
- Participation is voluntary
- All participants will need to sign a standard consent form
- Interviews will be recorded with your consent
- Your individual information will be kept confidential
- Beren Therapeutics will share a summary of non-identifiable insights with the NNPDF
05/01/2026
Niemann-Pick Type C Brain Imaging Research Study
Dr. Jaymin Upadhyay and his team at Boston Children’s Hospital are leading a research study to better understand NPC through advanced brain imaging and non-invasive assessments.
Who is eligible?
Patients diagnosed with Niemann Pick Type C (NPC) or healthy individuals.
What does the study involve?
During this study, we will ask you to complete questionnaires, cognitive tasks, motor tasks, give a blood sample, perform tasks via a functional near infrared spectroscopy (fNIRS) tool, undergo muscle integrity and healthy assessment through a non-invasive measure called electrical impedance myography (EIM), and undergo
MRI.
The study visit will last approximately 7 hours divided over two days. Participation in this research will not alter or interfere with any of the standard clinical procedures or treatment that one would normally receive if not partaking in this research. All research studies will take place at Boston Children’s Hospital or McLean Hospital.
What if I live out of town, can I still participate?
Yes! If an NPC patient lives outside of the Boston metropolitan area, our lab can help coordinate your visit to Boston or do a study visit in the comfort of your own home with our bed-side technologies. We will also provide reimbursement up to $1000 for travel-related expenses.
Will I get paid to be a part of this study?
An NPC patient or healthy subject who participates in this study visit will be paid $150. Those only undergoing bed-side assessments (no MRI) will be paid $75.
For study information, please contact:
Jaymin Upadhyay, PhD
917-736-1541
[email protected]
Raquel vanGool
617-301-0920
[email protected]
Additional Research Study Information
08/13/2025
ASMD Accelerate
Just over a year ago, Wylder Nation and NNPDF partnered together to launch ASMD Accelerate to support the ASMD research community. To date, we’ve had 15 contribute their child’s de-identified medical data in support of ASMD research!
If you are interested in joining ASMD Accelerate and making a difference in the future of ASMD research, we are still enrolling new members.
This research study is a way your child’s journey can contribute to ASMD research without the need for travel, lab testing, or physical record collection coordination. Additionally, if your child’s journey with ASMD has ended this study can help carry on their legacy and contribute to improving the future of ASMD care.
Signing up for ASMD Accelerate takes less than 10 minutes and PicnicHealth will do the hard work of collecting your child’s records on your behalf. Let us know if you have any questions!
Visit picnichealth.com/asmd-wylder-nation or email [email protected] with any questions.
NPC Quality of Life Research Project
Important research project into quality of life in people with NPC.
NNPDF is reaching out to our NPC Community with a request for help with a PhD research project exploring how we measure quality-of-life in people living with NPC.
Jackson Pountney is a PhD student at Aston University in Birmingham, UK, who is undertaking this research. For this project to be successful, we need as many NPC patients and families as possible to fill in some questionnaires that look at quality-of-life and general health. They should take about 20 minutes to complete.
This research is being supervised by Dr. Rebecca Knibb at Aston University. Dr. Knibb is an Associate Professor in psychology and has a history in Niemann-Pick disease research.
The work is funded by Niemann-Pick UK, the National Niemann-Pick Disease Foundation, the International Niemann-Pick Disease Alliance, and the International Niemann-Pick Disease Registry.
You can help by completing questionnaires in the INPDR Patient Reported Database (PRD). By joining the PRD, not only will you provide data for Jackson’s PhD, but you will also be contributing data that can support other research projects around the world.
Visit registry.inpdr.org/insight/prd/ or scan the QR code to join. Learn more about this project.
By having as many community members joining the PRD and completing questionnaires by this date, we can ensure this project is successfully completed.
If you have questions about Jackson’s project please email [email protected] and if you have questions about the PRD, please email [email protected].
Thank you for your support.