Why aren’t there treatments for CMT yet?

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

In this CMT Research Foundation video series, we’ve answered the most frequently asked questions about CMT treatments and the search for a cure. Watch as leading experts in research and drug development discuss the challenges of bringing an effective therapy to market and what the patient community can do now to accelerate treatments and cures.

New CMT Researcher Video Series

Demystifying the Drug Development Process

In this video, leading neurologists and geneticists discuss discoveries in the pipeline. The conversation also explores how patient involvement can help speed the progress of drug development.

Experts Featured

  • Chris Austin, Director, former, 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 underwriting this video.

AcuraStem logo

How Patients Can Change the Pace of Drug Development

Experts from DTx Pharma and Cure SMA discuss the influence patients and patient-led organizations can have on the drug development process. Up until recently, spinal muscular atrophy (SMA), also a genetic neuromuscular disease, had no treatments or cures, but now has its first FDA-approved treatments. The CMT Research Foundation is encouraging pharmaceutical companies to apply learnings in SMA (and other related diseases) to speed progress in CMT. Patients can also make a significant contribution.

Experts Featured

  • Dr Jill Jarecki, Chief Scientific Officer, CureSMA
  • Raffaella Gesuete, Scientist, DTx Pharma
  • Gary Donaldson, patient living with CMT1X

Thank you to our partners at Cellatoz Pharmaceuticals and ToolGen for underwriting this video.

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Cellatoz Therapeutics logo

How We Get from Today to FDA-Approved Treatments

Foundations working on other diseases like ALS, spinal muscular atrophy, and multiple sclerosis helped bring treatments to market. How did they get the FDA to approve those drugs? What is the outlook on gene therapy approval for CMT? And how can patients expedite the process?

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 underwriting this video.

Conversations with Researchers Working in CMT

We sat down one-on-one to have conversations with leading scientific experts in CMT to hear about their latest projects and when patients will see tangible progress.



Hear About New Videos & CMT Research Updates

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