How does the drug development process work, and why does it take so long?

Why aren’t there treatments for CMT after all this time, and when can we expect them?

In our new CMT Research Foundation video series, we’re asking and answering your most pressing questions about CMT treatments and the road to cures. Watch as leading experts in research and drug development take on often-avoided questions and shine a spotlight on what the patient community can do to accelerate treatments and cures now.

New CMT Researcher Video Series

Episode 1: Demystifying the Drug Development Process

In this video, leading neurologists and geneticists will have you on the edge of your seat as they share when patients can expect treatments for CMT and discuss discoveries in the pipeline. The conversation also explores how patient involvement can be one of the “prime disruptive technologies” to make the greatest progress in drug development.

Experts Featured

  • Chris Austin, Director, National Center for Advancing Translational Sciences, National Institutes of Health
  • Sindhu Ramchandren, Medical Director — Neurology, PRA Health Sciences

Thank you to our partners at AcuraStem and Shift Pharmaceuticals for sponsoring this video and underwriting the costs to make it possible.

AcuraStem logo

Episode 2: How Patients Can Change the Pace of Drug Development

In this video, experts from DTx Pharma and Cure SMA discuss the influence patients and patient-led organizations can have on the drug development process when they get involved and challenge conventions. Up until recently, spinal muscular atrophy (SMA), also a genetic neuromuscular disease, had no treatments or cures. Within the past five years, SMA now has its first FDA-approved treatments. You’ll hear how the CMT Research Foundation is initiating innovation in drug development by encouraging pharmaceutical companies to apply learnings in SMA (and other related diseases) to speed progress in CMT. Yes, patients can make all the difference.

Experts Featured

  • Dr Jill Jarecki, Chief Scientific Officer, CureSMA
  • Raffaella Gesuete, Scientist, DTx Pharma
  • Gary Donaldson, Community Manager, CMT Research Foundation and patient living with CMT1X

Thank you to our partners at Cellatoz Pharmaceuticals and ToolGen for sponsoring this video and underwriting the costs to make it possible.

Cellatoz Logo
Cellatoz Therapeutics logo

Episode 3: How We Get from Today to FDA-Approved Treatments

We’ve watched as other diseases like ALS, spinal muscular atrophy and multiple sclerosis have brought treatments to market. Yet, no drugs are currently approved to treat CMT. What does it take for the FDA to approve a drug? What’s the outlook on gene therapy approval for CMT? And how can patients expedite the process? In this video, Dr. Peter Marks from the FDA will answer these questions and more, including what we need in CMT to bring a drug to market.

Expert Featured

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

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

Conversations with Researchers Working in CMT

We sat down one-on-one to have intimate conversations with leading scientific experts in CMT to hear the latest on the projects they’re working on and the progress patients can expect.



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