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.
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).
Pharnext announces new positive results from the ongoing open-label extension study of their drug PXT3003
Applied Therapeutics, Inc. has announced that their drug candidate, AT-007 (Govorestat), has received orphan medicinal product designation
The study highlights the significant impact of neuropathic pain on the quality of life and psychosocial well-being of individuals with CMT.
Mitochondria are the powerhouses of our cells. Think of them as our body’s batteries. Mitochondrial disease causes these batteries to run low.
HNF TRIAD Academic Partner Connecticut Children’s Publishes Results of CMT Pediatric Natural...
Hereditary Neuropathy Foundation Re-Launches One-of-A-Kind Patient Registry for...
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