CMT Kenneth Raymond

CMT Highlight Reel: How does CMT data drive research using GRIN?

by | Mar 15, 2023 | 1 comment

Kenneth Raymond provides an overview of why sharing CMT symptoms in GRIN is so important!

The Global Registry for Inherited Neuropathies was created by HNF to address the issue of the lack of quantitative and structured data collected on CMT disease. The registry collects private, de-identified data that is valuable for research, particularly for biotech and pharma. The registry helps researchers to gauge the effectiveness of CMT drugs by providing a large window of symptom data. The prevalence rates are important for CMT researchers to have a sufficient patient population for trials. Ultimately, the registry helps to advance our understanding of CMT and will help advance drug developments.

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HNF is Reshaping Drug Repurposing for CMT

HNF is Reshaping Drug Repurposing for CMT

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.

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1 Comment

  1. Barbara Collins-Newing

    I would love to help research into CMT but live in Southern England and have problems with walking and driving any great distance. Is there anything I can do to help?


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