Could you be 1 of the estimated 3300 patients in the US with the gene causing SORD Deficiency? You may have been diagnosed with CMT2, or distal hereditary motor neuropathy (dHMN). Join us as we learn more about SORD Deficiency and an investigational NEW Treatment with Shoshana Shendelman, Founder and CEO of Applied Therapeutics.
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?
As we start the new year energized and ready to continue our mission of bringing treatments and...
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.
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
0 Comments