COMBINEDBRAIN HNF

CMT Biobank: We Need Your HELP!

by | Feb 1, 2024 | 0 comments

CMT Biobank: We Need Your HELP!

HNF is excited to continue collecting samples for researchers and industry to help develop treatments for CMT. CMT impacts the quality of life starting in childhood and is progressively debilitating. Currently, there are no treatments, but there are many potential therapies in the pipeline. There are still gaps in understanding the natural history of the disease correlation of genotype/ phenotype, the availability of patient biospecimens for translational research, and the validation of drug candidates and biomarkers for CMT.

HNF is the only CMT advocacy group that gives access to these patient samples at minimal cost.

The locations for specimen collection are currently being discussed. Please give us feedback if you would like us to come to a city near you!

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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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