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.
HNF’s website provides CMT community, academia, and biotech industry a hub for exploring research, clinical trials, vital resources, and ways to get involved.
HNF, in partnership with Rarebase, is leading the charge in the first-ever research initiative to tackle multiple types of CMT in one project using its tech-enabled drug discovery platform called “Function.”
Without your participation, researchers won’t have the essential patient information to develop drugs, gene therapies, and clinical trials for Charcot-Marie-Tooth and other Inherited Neuropathies. In addition, as GRIN grows, we gain greater insights from you as patients to help accelerate therapies for Charcot-Marie-Tooth (CMT) and Inherited Neuropathies.
A study of the correlation between gait abnormalities, activity monitoring parameters, CMTPedS and a biomarker in children with Charcot-Marie-Tooth disease.
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