News & Stories

See the latest news about CMT drug development and read stories from the CMT community that highlight why we must deliver treatments and cures during our lifetime.

One Million Steps to the Holidays for CMT

By: Gary Donaldson, community manager, CMT Research Foundation As I approach my one-year anniversary as the community manager for the CMT Research Foundation, I’ve thought a lot about how I should mark this occasion. I decided the best way to celebrate is by facing...

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How We Get from Today to Approved CMT Treatments: An Interview with FDA Director Dr. Peter Marks

How We Get from Today to Approved CMT Treatments: An Interview with FDA Director Dr. Peter Marks

The CMT Research Foundation is asking and answering the most pressing questions patients have about the need for treatments and cures. In episode 3, CMT Research Foundation CEO Susan Ruediger interviews the FDA’s Dr. Peter Marks to discuss gene therapies as a potential treatment option for CMT, what the approval process looks like and how patients can expedite it.

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WATCH: Power of the Patients: How We Change the Pace of Drug Development

The CMT Research Foundation is asking and answering the most pressing questions patients have about the need for treatments and cures. In episode 2, CMT Research Foundation CEO Susan Ruediger interviews Cure SMA’s Chief Scientific Officer Dr. Jill Jarecki, DTx Pharma Scientist Dr. Raffaella Gesuete and patient advocate Gary Donaldson to understand what patients can do today to influence the drug development process and speed progress. {HINT: Patients have the power to change everything!}

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