First Ever Biorepository For Charcot-Marie-Tooth, HNF Launches The CMT Biobank
The Hereditary Neuropathy Foundation (HNF), an advocacy and research 501c3 non-profit, today...
The Hereditary Neuropathy Foundation (HNF), an advocacy and research 501c3 non-profit, today...
Dtx today announced that the FDA has granted Orphan Drug Designation to DTx-1252, an investigational FALCON small interfering RNA (siRNA) therapeutic for the treatment of Charcot-Marie-Tooth Disease Type 1A (CMT1A).
Applied Therapeutics, Inc. has announced that their drug candidate, AT-007 (Govorestat), has received orphan medicinal product designation
How do we honor the inevitable grief that comes with disability and establish healthy boundaries...
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?
She is at it, again! Jenny Decker’s “Just a Lap” circumnavigating the globe In 2016, Jenny kayaked...
HNF’s website provides CMT community, academia, and biotech industry a hub for exploring research, clinical trials, vital resources, and ways to get involved.
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