Impact That Matters
Learn about HNF’s Research InitiativesResearch programs that can change people’s lives
We prioritize research programs that will change people’s lives by partnering with innovative researchers and industry leaders.
We ONLY support projects that have potential to have an impact on human health.
- To partner, contact Joy Aldrich: [email protected]
- To submit a research proposal, contact Allison Moore: [email protected]
TRIAD
HNF developed the Therapeutic Research in Accelerated Discovery (TRIAD) as a patient-led collaborative network with academia, government, and industry to develop treatments for CMT. Currently TRIAD involves many groups that span the drug discovery, drug development, and diagnostics continuum.
Our core philosophy mandates that we collaborate with our patient community and strategic partners compassionately, honestly and with integrity. The patient always comes first and drives our patient support and research efforts.
CMT & Pediatrics
HNF has a dedicated CMT & Pediatric initiative to support the development of clinical trials.
GRIN
As part of TRIAD, in 2013, the Global Registry for Inherited Neuropathies (GRIN) was developed as an extension to conduct patient-focused research & development for treatments and cures. The patient voice is at the forefront of all we do. By Incorporating the patient voice from the very beginning, our research programs have the greatest potential for success.
HNF funds research with one goal in mind, advancing to clinical trials. Partnerships vary depending on research goals, milestones met, preclinical or clinical development and the strategic alliance agreement.
Research Opportunity: CMT-SORD
Research Opportunity: CMT-SORD I am writing to you today because there is a research opportunity you may be eligible for. When you joined HNF’s Global Registry for Inherited Neuropathies (GRIN), you agreed to be contacted regarding these opportunities. Since 2001, the...
Digital Health Technologies for Charcot-Marie-Tooth (CMT) Disease
Digital Health Technologies for Charcot-Marie-Tooth (CMT) DiseasePatients attending the Clinical Trial Readiness Summit will be eligible to participate in this two-week study. All participants will join the clinical researchers and HNF staff for a private dinner on...
Breaking News for SORD Deficiency Patients
The INSPIRE trial is a Phase 3 double-blind placebo-controlled registrational study evaluating the effect of once-daily (QD) oral govorestat (AT-007) in 56 patients aged 16-55 with SORD Deficiency in the US and Europe.
Congratulations to GRIN Advisory Board member, Professor Joshua Burns
Congratulations to GRIN Advisory Board member, Professor Joshua Burns, for being recognized by the Australian Academy of Health and Medical Sciences for his outstanding contributions to medical and health sciences.
Great News From HNF TRIAD Industry Partner — Pharnext
Pharnext announces new positive results from the ongoing open-label extension study of their drug PXT3003
Great News From HNF TRIAD Industry Partner — Applied Therapeutics
Applied Therapeutics, Inc. has announced that their drug candidate, AT-007 (Govorestat), has received orphan medicinal product designation
Cambridge-led natural history study identifies MTRFR/C12orf65 deficiency to improve diagnosis and therapy development
This research will potentially show a method that may be quicker and better at identifying baseline disease phenotypes to support research and clinical trials. Cambridge-led retrospective natural history study funded by the Hereditary Neuropathy Foundation identifies...
Pharnext unveils the latest progress of the PREMIER Phase III clinical trial for CMT1A
The PREMIER trial, initiated in March 2021, is an international, randomized, double-blind, two-arm placebo-controlled, pivotal Phase III study, where the primary objective is to evaluate the efficacy and safety of PXT3003 versus placebo in mild-to-moderate CMT1A...
HNF is Reshaping Drug Repurposing for CMT
HNF has partnered with Rarebase, a public benefit precision medicine company that has screened a large library of FDA approved small molecules to identify candidates for various types of CMT. Their tech-enabled drug discovery platform is called Function™. There are many published discoveries on the genetic cause of many types of CMT, including an understanding of the basic mechanism of disease and potential targets for FDA-approved drug repurposing. It is this understanding that allows HNF and Rarebase to target the genetic root cause of CMT.
Hot off the Press: CMT Update
As we start the new year energized and ready to continue our mission of bringing treatments and cures for CMT, we have rebranded our CMT Update newsletter to feature our lead research programs including the GRIN patient registry, the HNF-funded research milestones...