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.
Mandos Health Market Research for NPC Patients
NNPDF would like to make the NPC Community aware of the opportunity to share your experience and participate in market research sponsored by Mandos Health by Beren Therapeutics.
The purpose of this market research is to better understand the experience and needs of NPC patients who are currently taking commercially approved therapies and their caregivers to help inform the development of a patient support service program for a potential new therapeutic option. Mandos Health by Beren Therapeutics is seeking participants for one-on-one interviews to share their perspectives.
Study Details:
- Format: Individual 60-minute interview via Zoom
- Scheduling: Flexible timing based on your availability
- Participant Incentive: As a thank you for your participation, each participant will receive a $150 gift card
Who can participate?
All NPC patients must currently be on a commercially approved therapy (Miplyffa and/or Aqneursa) AND fit one of these three categories:
- Category 1: Patients who are currently on Miplyffa and/or Aqneursa AND have never participated in any EAP* including adrabetadex
- Category 2: Patients who are currently on Miplyffa and/or Aqneursa AND have participated in the adrabetadex EAP*
- Category 3: Patients who currently on Miplyfaa and/or Aqneursa AND have transitioned from an EAP* other than the adrabetadex EAP
*What is an EAP? An Early Access Program (EAP) allowed the NPC patient to receive the medication free of charge after completing the clinical trial but before FDA approval.
How to indicate interest: If you are an interested patient or caregiver or a patient described above and are interested in participating, or would like more information, please reach out directly to : Rochelle Ardesher at [email protected].
In your email, please indicate which Category (1,2,3) you fulfill based on the above criteria. The company is looking to speak with up to 10 individuals. Qualifying participants will be selected on a first come, first served basis.
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
- Mandos Health by Beren will share non-identifiable insights with the NNPDF
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.