In June 2013, HNF made an important decision to sharpen the organization’s mission. We now place greater emphasis on initiatives that take our research discoveries and move them into the next phase of the drug-development process.

We organize our research into three areas:

  • Basic Research: Work spanning academic institutions around the world through government agencies such as the CDC.
  • Translational Research: The BioPontis Alliance is helping us advance early-stage assets into potential clinical trial candidates.
  • Clinical Research: We are working with Pharnext and Quest Diagnostics to support clinical research and ensure early, accurate diagnosis, supported by the Global Registry for Inherited Neuropathies (GRIN).

We would like to do so much more. We have identified two inherited neuropathies, CMT6 and Autosomal Dominant Optic Atrophy, that can cause blindness at a young age, and we need additional funding to help scientists develop screens and fill the gaps in knowledge.

Predicting the future is never certain, but in 2015 we hoped to see the first Phase III trial of a drug for CMT1A, which would mark a major milestone for the CMT1A community. We worked hard through 2014 to support Pharnext in that goal and continued to do so. Confirming patient diagnoses and recruiting participants to GRIN remained essential to giving those trials the best chance of success.

HNF has not stopped working for you, the patients, caregivers, and advocates, and we pledge we will not give up the fight to find viable treatments for this disease. The scientists we fund are dedicated people working collaboratively toward the day when effective treatments will be available to you and your family. CMT is truly a family disease. It touches everyone in your life, and HNF takes that reality to heart. There is finally light at the end of the tunnel.