Early preclinical research indicates that ALE2 could effectively slow or halt the progression of these debilitating symptoms, offering new hope for patients with these rare forms of CMT.
HNF is thrilled to announce that we have been accepted to host a 1-hour panel discussion during the Bio International Convention in San Diego, CA, in June 2024.
Pediatric CMT Natural History Study enhancement to their Charcot-Marie-Tooth (CMT) and Inherited Neuropathies (IN) patient registry, Global Registry for Inherited Neuropathies (GRIN).
This year, HNF is spotlighting 3 Key Pillars vital to CMT Awareness & Research success.
We invite you to choose from each pillar throughout this September to drive Impact That Matters!
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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