What is CMT-SORD?

In 2019, with Charcot-Marie-Tooth Association (CMTA) funding, researchers discovered CMT-SORD. CMTA Alliance Partner, Applied Therapeutics, then quickly identified a drug which they successfully tested in a newly CMTA-developed CMT-SORD model before rapidly advancing into the current Phase III trial.

At the time of discovery in 2019 and published in 2020, CMTA Strategy to Accelerate Research (STAR) Advisory Board member, Stephan Zuchner, M.D., Ph.D., hypothesized that CMT-SORD could very well become the first truly treatable type of CMT. And we’re almost there!

Why do we need your help?

Supported by CMTA, Applied Therapeutics is asking for accelerated approval of their CMT-SORD drug and is preparing what is called a pre-NDA (pre-New Drug Application) meeting request with the U.S. Food and Drug Administration (FDA). This is the step in the FDA process where the pharmaceutical company presents data and makes its case for why the FDA should grant a new drug application (NDA).

CMT-SORD community member Vittorio Ricci started a petition and we need at least 1,000 signatures by May 1st.

Should the FDA move this drug into accelerated approval, not only will it help thousands of CMT-SORD community members, but it will set a precedent by which other potential CMT drugs could benefit. Granting Applied Therapeutics’ pre-NDA meeting for CMT-SORD has the real possibility of helping all CMT community members regardless of type.

Who can participate?

Anyone can take action and sign today!

Take Action!

Signing the petition today sends a strong and clear message to the FDA that CMT and the needs of the CMT community matter. Hope for tomorrow begins with your signature today!

Sign the Petition