Surveys, Studies, & Market Research
Current Survey, Studies, and Market Research Opportunities
- ASMD Accelerate
- Impact of NPC on Quality of Life
- NPC Quality of Life Research Project
- NPC Brain Imaging Research Study
Impact of NPC on Quality of Life
Share your experience and help to better understand the impact of Niemann-Pick type C (NPC) on Quality of Life
We know that Niemann-Pick type C (NPC) affects the quality of life for those living with the condition, as well as for their families and caregivers. Quality of life means how comfortable, happy, and capable you feel as you live your daily life.
To better understand the overall impact of NPC on quality of life, we would like to invite you to share your experience by participating in a research study.
The purpose of the study is to gain insight into the challenges faced by individuals affected by NPC, including the effects of symptoms, experiences with treatment and clinical care, and any unmet needs.
By participating in this research study, you will have the opportunity to share your unique experience, whether as someone living with NPC or as a caregiver.
The survey is open to those aged 18 years and over who:
- are fluent in English (including non-native English speakers) AND
- have a confirmed diagnosis of NPC OR
- are the parent or carer of a person with a confirmed diagnosis of NPC
The survey is completed online and should take no longer than 50 minutes to complete. It forms part of a larger study which includes interviews with individuals with NPC or their caregivers, exploring the questions in this survey in more depth. You do not have to take part in the interview if you do not want to.
Everyone who completes the survey will have the opportunity to be entered into a prize draw to win a £100 voucher.
With new therapies recently approved in the USA and ongoing clinical trials, understanding the views of the NPC community is more important than ever.
By sharing your experience, you will help NPUK and its research partners better understand the true impact of NPC on Quality of Life (QoL). Your insights will play a vital role in shaping how we communicate these challenges to healthcare providers and policymakers, advocating for improvements in care, support, and access to treatments for the Global NPC community.
What happens next
Click the link below to answer a few screening questions, which will help tailor the survey to you. You will then be directed to your participant information leaflet to learn more about the study and complete the consent form before starting the survey.
If you have any questions, please contact [email protected], [email protected] or [email protected].
01/03/2025
Niemann-Pick Type C Brain Imaging Research Study
NNPDF has been asked to share the following information regarding a Niemann-Pick Type C Brain Imaging Research Study at Boston Children’s Hospital under the research group of the Division of Genetics and Genomics, Department of Medicine, and Department of Anesthesiology, Critical Care and Pain Medicine.
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 nonvasive measure called electrical impedance myography (EIM), and undergo MRI.
The study visit will last approximately 3-4 hours.
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 $500 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.
How can I get more information?
For more information please call 917-736-1541 or email Jaymin Upadhyay, PhD at [email protected]
Additional Research Study Information
08/20/2024
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.
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.
