Big News for the CMT-SORD Community

Updates from HNF’s TRIAD Partner, Applied Therapeutics

In a newly released press release, Applied Therapeutics, a valued HNF TRIAD partner, shared exciting progress in the development of treatments and diagnostics for CMT-SORD, a rare and often under diagnosed form of Charcot-Marie-Tooth disease.

Treatment Progress: Govorestat Heads Toward FDA Review

Applied Therapeutics is preparing to meet with the FDA this quarter to discuss the next steps toward a potential approval of Govorestat, a treatment developed specifically for people living with CMT-SORD.

At the Peripheral Nerve Society 2025 Annual Meeting, new data from their INSPIRE trial showed that Govorestat slowed the progression of the disease, improved mobility, and was well-tolerated by participants.

Free Urine Testing for CMT-SORD Now Available

To help more individuals get diagnosed, Applied Therapeutics has launched a FREE urine-based Sorbitol Assay to identify potential cases of CMT-SORD.

Want to learn more? Contact HNF for details on how to access this free testing.

Email us at our contact form.

More Rare Disease Research on the Horizon

Applied Therapeutics is also advancing work on:

  • PMM2-CDG, with new data to be presented this October at the American Society of Human Genetics (ASHG) Annual Meeting
  • Classic Galactosemia, with continued regulatory discussions underway

HNF’s TRIAD (Therapeutic Research in Accelerated Discovery) program brings together patient voices, researchers, and industry leaders like Applied Therapeutics to fast-track treatments for rare diseases like CMT.

Click here to read the full press release.

Thank you for being part of this journey.

Together, we’re turning hope into action.