Presented by: Rarebase
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.” Rarebase will screen a compound library of thousands of FDA-approved drugs and novel drugs, targeting ten subtypes of CMT. Join Rarebase, Founder & COO Omid as we dive deeper into this exciting new technology and what it means for treating CMT.
Pharnext announces new positive results from the ongoing open-label extension study of their drug PXT3003
Applied Therapeutics, Inc. has announced that their drug candidate, AT-007 (Govorestat), has received orphan medicinal product designation
The study highlights the significant impact of neuropathic pain on the quality of life and psychosocial well-being of individuals with CMT.
Mitochondria are the powerhouses of our cells. Think of them as our body’s batteries. Mitochondrial disease causes these batteries to run low.
HNF TRIAD Academic Partner Connecticut Children’s Publishes Results of CMT Pediatric Natural...
Hereditary Neuropathy Foundation Re-Launches One-of-A-Kind Patient Registry for...
Kenneth Raymond provides an overview of why sharing CMT symptoms in GRIN is so important!
Interested in driving CMT research forward? Watch our new 7-min GRIN Demo video explaining the steps in creating and updating your GRIN account!
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?
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