CMT&Me is an observational, non-interventional study, sponsored by Pharnext, to collect Real-World Evidence (RWE) from people living with CMT. The objective of the study is to better understand the impact of the disease on patients’ daily lives: the burden of CMT, its natural history and treatment, and medical, social and pharmacoeconomic effects.
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).
HNF is proud to present “CMT Presents,” a virtual presentation series designed to spread awareness in the communities, companies, and groups closest to you!
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.
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