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Meet virtually with host, Julie Stone & fellow CMTers from all over the world for a fun and...
HNF is proud to present “CMT Presents,” a virtual presentation series designed to spread awareness in the communities, companies, and groups closest to you!
How do we honor the inevitable grief that comes with disability and establish healthy boundaries...
Kenneth Raymond provides an overview of why sharing CMT symptoms in GRIN is so important!
Charcot-Marie-Tooth disease is one of several hereditary neuropathies that affect the peripheral nervous system. CMT affects about 1 in every 2,500 people, making it one of the most common inherited neurological disorders worldwide.
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.”
Clinical Trial Update for SORD Deficiency – Applied Therapeutics – Dr. Shoshana Shendelman
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.