Total posts in Category: 11
CMT Summit + Retreat 2025: Uniting Patients, Researchers, Regulators, and Industry Leaders to...
CMT Simplified is here to make staying informed about Charcot-Marie-Tooth disease (CMT) easier...
Kenneth Raymond provides an overview of why sharing CMT symptoms in GRIN is so important!
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.
How do we know if our symptoms are Charcot-Marie-Tooth Disease related, and how can we drive research to better answer this question?
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.
Could you be 1 of the estimated 3300 patients in the US with the gene causing SORD Deficiency?
Hear from Allison Moore, Founder/CEO of the Hereditary Neuropathy Foundation about the past, present and future or research.
Join us for this webinar as we learn more about Sorbitol Dehydrogenase (SORD) Deficiency
CMT&Me is an observational, non-interventional study, sponsored by Pharnext, to collect Real-World Evidence (RWE) from people living with CMT.
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