Presented by: Rarebase
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.” Rarebase will screen a compound library of thousands of FDA-approved drugs and novel drugs, targeting ten subtypes of CMT. Join Rarebase, Founder & COO Omid as we dive deeper into this exciting new technology and what it means for treating CMT.
Seeking Participants for a New CMT Clinical Trial: A Chance to Help Advance Treatment If you’re...
Join the thousands of HNF volunteers who are fueling CMT research. The more you share, the more we’ll learn. Complete your GRIN surveys today!
HNF Awards the inaugural HNF Clinical Translation Fellowship in the amount of $170,000 to Kayla...
Do you want to make a difference in CMT research? HNF is looking for patients with a confirmed CMT diagnosis to participate in the CMT Biobank
The INSPIRE trial is a Phase 3 double-blind placebo-controlled registrational study evaluating the effect of once-daily (QD) oral govorestat (AT-007) in 56 patients aged 16-55 with SORD Deficiency in the US and Europe.
HNF Contributes CMTRN Data to C-Path’s RDCA-DAP, Strengthening Research Capabilities for Inherited...
HNF is thrilled to announce that we have been accepted to host a 1-hour panel discussion during the Bio International Convention in San Diego, CA, in June 2024.
HNF is excited to continue collecting samples for researchers and industry to help develop treatments for CMT.
Hereditary Neuropathy Foundation Partners with InformedDNA® on CMT Genie Project
In July 2023, Novartis announced the acquisition of DTx Pharma, a San-Diego based, preclinical stage biotechnology company focused on leveraging its proprietary FALCON platform to develop siRNA therapies for neuroscience indications.
I wanted to let you know I have HNPP and have been suffering with sever neck, shoulder and upper arm muscle pain since 2011. Nothing has relieved this pain. I’ve done everything possible. Then I started having GI problems, sweating, nausea, vomiting, cramps after eating and have passed out twice. So went through all GI and Endocrinology testing. The last test was a Hida Scan. I was give CCK the last 30 minutes of the test. When I got off the table I had no pain! I felt like a new person. No GI symptoms and this lasted 11 days and slowly the pain and symptoms are coming back. So I researched CCK and found it was once a clinical trial for nerve pain and then I looked into the vagus nerve. If the nerve is damaged can cause all these symptoms especially GI and neck, should and muscle pain. All my tests from GI and Endo were normal and GI doctor agreed that it is the vagus nerve. I don’t know how CCK could be taken orally, by I’m seeing the Endo doctor, who is really interested in this, to see what can be done. I feel CCK was a miracle and for 11 days I felt like I had a new life. Very depressing that the symptoms have started again. I wish HNF would look into patients receiving CCK who have severe chronic pain and also GI symptoms. Something is not working correctly. Advised to see Neurosurgeon. Living with chronic severe pain and GI symptoms is like not living at all! Please look into this. Thank you.