Overview

Compassionate Use of Triheptanoin (C7) for Inherited Disorders of Energy Metabolism

Status:
No longer available
Trial end date:
1969-12-31
Target enrollment:
Participant gender:
Summary
This is a compassionate use study to allow patients already taking triheptanoin (C7) through previous studies to continue to receive the supplement. It will also allow triheptanoin supplementation in patients with qualifying disorders if they are failing conventional therapy.
Details
Lead Sponsor:
Jerry Vockley, MD, PhD
University of Pittsburgh
Collaborator:
Ultragenyx Pharmaceutical Inc