For INAD Patients and Families

What has happened:  A 2015 research publication by Retrotope collaborators on PLA2G6 associated neurodegeneration (PLAN) ( provided a rationale that Retrotope’s drug candidates against lipid peroxidation damage might provide a new treatment paradigm for Infantile Neuroaxonal Dystrophy (INAD). In October 2017 the FDA granted orphan designation for RT001 in the treatment of PLAN, which includes INAD. Orphan designation does not authorize a clinical trial; clinical trials require an Investigational New Drug (IND) application to be approved by the FDA. On March 26, 2018, encouraged by preliminary results in Expanded Access (Compassionate Use) patient trials in two INAD children during 2017, Retrotope filed an IND application with the FDA for a formal clinical trial in INAD. Once initiated, full details will be available from the FDA website and from the company.

What to expect:  Retrotope is awaiting FDA approval to launch a late stage US-based clinical trial of its compound, RT001, in children with classical INAD. Since the trial may begin shortly, and the number of slots for enrollment (12 to 15) are limited, Retrotope has brought on a dedicated Patient Coordinator, Sarah Endemann.  Sarah brings over a decade of experience in working closely with patients and their families as a certified clinical research coordinator in Southern California, and is herself the mother of three young children.  Sarah will be the principal interface to INAD families and will be able to review the trial protocol, inclusion and exclusion criteria, frequency of visits (over a 13 month period) and so on. If you would like to consider participating in the upcoming trial, feel free to contact Sarah by phone at 619-206-5944 or via email (, in advance of our formal announcement of the trial. If you have already contacted us, do not worry.  Sarah will be in touch shortly.  We are excited about what we have observed so far via Compassionate Use experience and look forward to working with as many families as possible in the near future.