Unwilling to accept the slow pace of progress in Charcot-Marie-Tooth (CMT) drug development, the CMT Research Foundation’s new video series asks and answers the most pressing questions about CMT treatments and how we can deliver them faster.

In this third episode, CMT Research Foundation CEO Susan Ruediger interviews Dr. Peter Marks, the director of the Center for Biologics Evaluation and Research at the Food and Drug Administration (FDA). His department is responsible for regulating and approving complex treatments, including gene therapies, which could transform the lives of CMT patients and their families.

Although other rare diseases like ALS, spinal muscular atrophy and multiple sclerosis have FDA-approved treatments, no drugs are currently approved to treat CMT. Susan and Dr. Marks discuss what it takes for the FDA to approve a drug, the promise of gene therapies for CMT and how patients can expedite the process. Watch now.


After watching this video, you will have answers to these questions:

  • What is the FDA’s top priority for approving drugs?
  • Why are gene therapies an important potential treatment option for CMT?
  • How many gene therapies are currently approved in the United States?
  • How does the FDA drug approval process work?
  • How can CMT patients expedite the process and be partners in progress?


Meet Dr. Marks

Dr. Peter Marks

Peter Marks, M.D., Ph.D., Director, Center for Biologics Evaluation and Research, Food and Drug Administration

Dr. Peter Marks received his graduate degree in cell and molecular biology and his medical degree at New York University and completed Internal Medicine residency and Hematology/Medical Oncology training at Brigham and Women’s Hospital in Boston. He has worked in academic settings teaching and caring for patients and in industry on drug development. He joined the FDA in 2012 as Deputy Center Director for the Center for Biologics Evaluation and Research and became Center Director in January 2016.

Thank you to our partner DTx Pharma for sponsoring this video and underwriting the costs to make it possible.

DTx Pharma

See More Videos

You can see all the videos in this series and what’s coming up here. Be the first to hear about new videos, see previews before they’re launched, and get access to newly released interviews by signing up to receive email notifications.