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.
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.
Congratulations to GRIN Advisory Board member, Professor Joshua Burns, for being recognized by the Australian Academy of Health and Medical Sciences for his outstanding contributions to medical and health sciences.
Pediatric CMT Natural History Study enhancement to their Charcot-Marie-Tooth (CMT) and Inherited Neuropathies (IN) patient registry, Global Registry for Inherited Neuropathies (GRIN).
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).
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