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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Great News From HNF TRIAD Industry Partner — Pharnext
Pharnext announces new positive results from the ongoing open-label extension study of their drug PXT3003
Great News From HNF TRIAD Industry Partner — Applied Therapeutics
Applied Therapeutics, Inc. has announced that their drug candidate, AT-007 (Govorestat), has received orphan medicinal product designation
Using Medical Cannabis for Managing Pain in Charcot-Marie-Tooth Disease — Patient Reported Outcomes
Data regarding medical cannabis use for pain relief in CMT from HNF’s CMT Patient Registry, GRIN
9 Key Findings from Your GRIN Surveys (and why you need to come back and complete more)
The study highlights the significant impact of neuropathic pain on the quality of life and psychosocial well-being of individuals with CMT.
HNF sheds Light & Research Funding on Mitochondrial Diseases Linked to CMT
Mitochondria are the powerhouses of our cells. Think of them as our body’s batteries. Mitochondrial disease causes these batteries to run low.
CMT Research Update: Pediatric Ankle And Gait Function
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 Charcot-Marie-Tooth Disease Research
Hereditary Neuropathy Foundation Re-Launches One-of-A-Kind Patient Registry for...
CMT-Associated Genes and Their Related Subtypes: The Definitive Guide
Kenneth’s groundbreaking book, CMT-Associated Genes and Their Related Subtypes: The Definitive...
Charcot-Marie-Tooth Virtual Support Group
The support group offers time to participate in a variety of both educations & open discussion meant to help everyone engage with others on similar journeys.
emBRACE it™ Episode #43
How do we honor the inevitable grief that comes with disability and establish healthy boundaries...
CMT Safe Space – Best Shoes for High-Arched Feet?
CMT Safe Space - Best Shoes for High-Arched Feet? Thanks to all those who joined last week! Check...
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