Impact
Twenty-five years of building what the CMT field did not have.
Since 2001, the Hereditary Neuropathy Foundation has helped build much of the groundwork the Charcot-Marie-Tooth community relies on today: among the first patient-led global CMT registries, a biorepository for CMT, a Patient-Focused Drug Development meeting that carried the patient voice to the FDA, and the patient-reported evidence that shapes how treatments are designed and reviewed. This is what that work has made possible, and where it goes next.
What "impact" means here
Impact, for HNF, is infrastructure other people can use. Not a single study, but the registry, the biorepository, the regulatory relationships, and the outcome measures that a treatment has to pass through to reach patients. GRIN, the Global Registry for Inherited Neuropathies, launched in 2013 and now tracks more than 100 CMT subtypes. The CMT Biobank links blood, DNA, and tissue to that data for qualified researchers.
We try to be careful with the word "first." Several of the milestones here are genuinely early examples of what they describe, and we qualify them where a broader claim would overstate it. The point is not the label. The point is that each piece lowers the distance between promising science and an approved treatment.
By the numbers
Explore our impact
Be part of the next first
Every milestone in this section was powered by people living with CMT who chose to share their story. Joining GRIN, the Global Registry for Inherited Neuropathies, is the most direct way to add yours.