HNF recently entered into a partnership with the French biopharmaceutical company, Pharnext, to help raise awareness of Charcot-Marie-Tooth (CMT) disease and support the CMT patient community through several initiatives. Building awareness is key! Pharnext’s support will assist HNF in distributing HNF’s CMT Update quarterly newsletter, enhancing the Global Registry for Inherited Neuropathies (GRIN), setting up activities for CMT September Awareness Month in the US and strengthening the CMT Inspire Community. Pharnext is an advanced clinical stage biopharmaceutical company discovering and developing new therapeutics that target multiple key disease pathways for orphan and common neurological diseases including Charcot-Marie Tooth disease. The company’s lead program PXT-3003 for the treatment of CMT 1A has completed Phase 2 clinical trials and pending discussions with the appropriate regulatory agencies will undergo Phase 3 clinical trials. PXT-3003 was recently granted Orphan Drug designation from the European Medicines Agency and the U.S. Food and Drug Administration. Learn more at www.pharnext.com or www.hnf-cure.org.
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.