Could you be 1 of the estimated 3300 patients in the US with the gene causing SORD Deficiency? You may have been diagnosed with CMT2, or distal hereditary motor neuropathy (dHMN). Join us as we learn more about SORD Deficiency and an investigational NEW Treatment with Shoshana Shendelman, Founder and CEO of Applied Therapeutics.
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).
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...
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