International Niemann–Pick Disease Alliance
(Available for download in Dutch, English, German,
Latin American Spanish, Mandarin, Persian, and Spanish)
Currently, we have enrolled 43 patients into the trial across 20 sites worldwide. We continue to screen and enroll patients. As a reminder, we need 51 patients to be enrolled in the trial. We thank you for your help and support so far, and we ask for your continued help and support in getting this trial fully enrolled.
If you know of anyone who is interested in participating in the trial, please contact Carrie Burke at carrie@vtessepharma.com or refer them to the nearest clinical trial site. A full list of clinical trial sites can be found at www.theNPCstudy.com.
The passage of the 21st Century Cures Act in the United States was due in no small measure to the activities of hundreds of patient advocacy groups and assorted other stakeholders that worked in overdrive the past two years pushing the bill to the finish line. Below are some of the provisions of the new law:
The act provides new funding over a 10-year period for initiatives that have the potential to significantly advance our understanding of disease and identify opportunities for new therapies.
The 21st Century Cures Act addresses the entire biomedical innovation system across discovery, development and delivery of new medical products. Here are examples of policy reforms in this act:
To find out more about the trial and to find a clinical trial site please visit www.theNPCstudy.com or https://clinicaltrials.gov/ct2/show/NCT02534844?term=vtesse&rank=1.