NNPDF Clinical Trial Pharmaceutical Survey – June 2016
In September of 2015, the following questionnaire was sent to CTD Holdings, Orphazyme and Vtesse by the National Niemann-Pick Disease Foundation (NNPDF) for their review, completion and comments. The intent was to:
a) collect the science behind the drug along with the trial details so that this information can be provided to family doctors and consultants who are treating patients with Niemann Pick Type C so that they can help guide patients who are interested in participating in a trial, and
b) to ensure that any pharmaceutical company wanting to run a trial on our children and young adults have done their due diligence in ensuring the safety of the drug and have done everything they can to ensure a favorable outcome.