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TRIADTherapeutic Research in Accelerated Discovery (TRIAD)

HNF developed the Therapeutic Research in Accelerated Discovery (TRIAD) in 2007 as a collaborative effort 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.

ucla          High content screens of 25,000 compounds for CMT1A (PMP22) and CMT1E (point mutation)


ucdavis           Developed CMT2A (MFN2 mutation) mouse model


shriners      Refined the MFN2 mouse model for distribution among the CMT research community


CMT1A assay (PMP22 mutation) developmentusc


burke         “Proof of concept” studies on an in vivo mouse model with CMT2A


Low content screens in zebrafish with CMT2A (MFN2)University_of_Sheffield_logo



WVU_logo      Established two colonies of transgenic CMT1A rats for testing various therapeutics in collaboration with CDC



Provided the CMT1A rat model to test various therapeutics




cdc     Established two colonies of transgenic CMT1A rats for testing various therapeutics in collaboration with CDC

pharnext   Partnering to support the development of the CMT patient community and to raise the awareness about CMT.


Cisbio        Developing a unique assay for CMT2A




quest       Collaborating to ensure early and accurate diagnosis for CMT

With research reaching such advanced stages we propose that some efforts should be made to fill the various gaps in clinical studies, in order to translate therapeutics to patients more rapidly.

We propose that in order to translate preclinical advances to the clinic and result in successful therapeutics there should be 1) alternative outcomes measures for CMT; 2) demonstration that potential treatments are having an effect on the QoL of patients; 3) genetic testing panels to facilitate identification of patients for future clinical trials; 4) use of the GRIN database to capture patients for future clinical trials and 5) education of clinicians and patients as to therapeutic options for these diseases.

We are addressing all of these areas and more with our TRIAD collaborators.


  1. Sames L, Moore A, Arnold RJG and Ekins S, Recommendations to enable drug development for inherited neuropathies: Charcot-Marie-Tooth and Giant Axonal Neuropathy, F1000Research, 3:83, 2014. http://f1000research.com/articles/3-83/v1
  1. Wood J, Sames L, Moore A, Ekins S, The multifaceted roles of rare disease parent / patient advocates in drug discovery, Drug Disc Today, 18: 1043–1051, 2013. http://www.drugdiscoverytoday.com/download/1215
  1. Beaulieu C, Samuels ME, Ekins S, McMaster CR, Edwards AM, Krainer AR, Hicks GG, Frey BJ, Boycott KM and MacKenzie, AE., A generalizable pre-clinical research approach for orphan disease therapy. Orphanet, 7(1):39, 2012. http://www.ojrd.com/content/pdf/1750-1172-7-39.pdf