CMT Awareness Month 2023 Hope. Courage. Action.
This year, HNF is spotlighting 3 Key Pillars vital to CMT Awareness & Research success.
We invite you to choose from each pillar throughout this September to drive Impact That Matters!
This year, HNF is spotlighting 3 Key Pillars vital to CMT Awareness & Research success.
We invite you to choose from each pillar throughout this September to drive Impact That Matters!
HNF is proud to present “CMT Presents,” a virtual presentation series designed to spread awareness in the communities, companies, and groups closest to you!
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?
0 Comments