HNF Video: Adaptive Driving Tour with Bernadette
CMT Highlight Reel: How does CMT data drive research using GRIN?
Kenneth Raymond provides an overview of why sharing CMT symptoms in GRIN is so important!
HNF Video: Adaptive Driving Tour with Bernadette
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.
The Social Security Administration (SSA) doesn’t make it easy for those with CMT to get the Social Security Disability Benefits they deserve.
Could you be 1 of the estimated 3300 patients in the US with the gene causing SORD Deficiency?
Joey’s family has a history of Charcot-Marie-Tooth, and he decided to get tested at the end of 2019. He learned at the age of 23 that he had CMT-1B and needed to adapt his way of life so that he could continue performing.
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