Side Menu Card

Arimoclomol Early Access Program for Niemann-Pick Disease

Arimoclomol Early Access Program for Niemann-Pick Disease

Contact

 If you are interested in participating in the study or want to learn more, please get in touch. Contact us
This study is now recruiting. Learn more about enrolling here.

Description
This study will provide early access to an investigational drug which does not yet have marketing authorization for human use, Arimoclomol, for a group of patients suffering from Niemann-Pick Disease Type C that are not eligible for or able to participate in other clinical trials.

What to expect
The study will involve the following procedures: (1) Administration of study drug (Arimoclomol)(2) Blood tests and urine tests(3) Medical history interviews(4) Questionnaire completion(5) Physical exams/height/weight(6) Review of medical records.

Related specialties
Smiling CHOP patient holding shopping bag

We need families like you

Your time and participation make a difference in supporting the work of the Research Institute and children in our community. Learn more about opportunities to participate in our research.