Niemann-Pick Type C Caregiver Preference Survey on Benefit Risk for Potential Treatments

Dear Friends in the NPC Community,

We invite families or caregivers of persons with Niemann-pick Type C disease to take a new survey that is designed to collect information about your priorities and preferences for treating Niemann-Pick Type C.

It is organized by the NPC Therapy Accelerator*.  In the survey, you will read about hypothetical treatments for Niemann-Pick Type C.  Some parts of the potential treatments described in this survey are based on real and investigational treatments and some are hypothetical.  The information collected here is important to help the U.S. Food and Drug Administration (FDA) understand your thoughts and feelings about potential treatments for NPC.

This survey is intended for parents and current and past caregivers of those with Niemann-Pick Type C. Those who have multiple children with Niemann-Pick Type C should complete one survey for each child and only one survey should be submitted for each child (e.g., two parents should not complete the survey separately on behalf of the same child). For parents/caregivers of older children or adults with Niemann-Pick Type C who are involved in their treatment decisions, you may consult with your child when responding.

Your participation in this survey is optional and if you start the survey, you can stop at any time.  The survey takes about 30 minutes.

Take the Survey

*About The NPC Therapy Accelerator

The NPC Therapy Accelerator (NTA) is a collaboration for the entire NPC community guided by the patient perspective.  Initiative members work with and gather relevant information from NPC companies and preclinical research groups to help them align their therapy development activities with the needs of the patient.  The NTA facilitates connections between biopharmaceutical companies and NPC scientists, labs, tools, and potential investors. Additionally, the NTA provides a forum for sharing data from the many initiatives ongoing in the NPC community.  The NTA will also work to facilitate access to healthcare for those impacted by NPC..

To start, the NPC Therapy Accelerator  will have a steering committee that includes:

  • Sean Kassen, Director, APMRF
  • Joslyn Crowe, Director, NNPDF
  • Chris Andrews, Parent; Firefly Fund,
  • Cristin Davidson, NPC Scientist
  • Phil Marella, Parent; Danas Angels Research Trust