NMD Pharma Initiates Phase 2 Study of NMD670 in Patients with Charcot-MarieTooth Disease Type 1...
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Clinical Trials
Total posts in Category: 21
HNF’s Wearable Tech Study Delivers Strong Results for CMT Clinical Trials
HNF’s Wearable Tech Study Delivers Strong Results for CMT Clinical Trials The Hereditary...
Seeking Participants for a New CMT Clinical Trial: A Chance to Help Advance Treatment
Seeking Participants for a New CMT Clinical Trial (for the US only): A Chance to Help Advance...
Digital Health Technologies for Charcot-Marie-Tooth (CMT) Disease
Digital Health Technologies for Charcot-Marie-Tooth (CMT) DiseasePatients attending the Clinical...
Breaking News for SORD Deficiency Patients
The INSPIRE trial is a Phase 3 double-blind placebo-controlled registrational study evaluating the effect of once-daily (QD) oral govorestat (AT-007) in 56 patients aged 16-55 with SORD Deficiency in the US and Europe.
Breaking News: HNF to Host “Revolutionizing Clinical Trials” Panel at Bio 2024
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 Enrollment Opening at Two Centers of Excellence
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 Pharma Receives FDA Orphan Drug Designation for DTx-1252 for the Treatment of Charcot-Marie-Tooth Disease Type 1A (CMT1A)
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).
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
CMT1A Clinical Trial Update: Pharnext PREMIER trial of PXT3003
The PREMIER Trial, which is being conducted in patients with mild-to-moderate CMT1A, is expected to enroll approximately 350 subjects ages 16-65 with a confirmed genetic diagnosis of CMT1A.
Accelerate Clinical Trials in Charcot-Marie-Tooth Disease (ACT-CMT) Calling CMT1A Patients Ages 18-75
This study is a new international research project funded by the National Institutes of Health (NIH) called Accelerate Clinical Trials in Charcot-Marie-Tooth Disease (ACT-CMT).
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