Impact That MattersLearn about HNF’s Research Initiatives
Research 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.
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.
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.
The CMT&ME app has updated the research app to include a brand new medical profile.
StarWise Therapeutics & HNF Form A Strategic Alliance to Bring a NextGen HDAC6 Drug Therapy to CMT2A (MFN2) Patients
HNF has been committed to funding HDAC inhibitor research since 2014.
Pharnext completed patient enrollment for the international Phase 3 clinical trial of PXT3003, Pharnext’s lead PLEODRUG ©, for treatment of CMT1A.
PARIS, December 18th, 2014 – Pharnext SAS today announced the proof of concept of its pleotherapy research and development approach based on a proprietary network pharmacology platform that identifies synergic combinations of drugs already approved for other diseases. Indeed, Pharnext’s lead pleodrug, PXT-3003, has shown positive results both in preclinical and Phase 2 clinical studies published today in the Orphanet Journal of Rare Diseases.
Research on CMT is global, and covers both laboratory and clinical studies. It is critically important to be aware of what is happening elsewhere as well as in the USA because it can have implications for what we do and fund at the HNF.
Some recent papers on Charcot-Marie-Tooth (CMT) disease go to show that we are steadily and impressively peeling back the complexity of the biology even though it is a relatively common rare disease with several thousand publications on it.