NMD Pharma Initiates Phase 2 Study of NMD670 in Patients with Charcot-MarieTooth Disease Type 1...
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Types of CMT
Total posts in Category: 1
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...
Jaxson’s Clayshoot for Mito CMTs
Jaxson’s Clayshoot for Mito CMTs Among the various forms of CMT, mitochondrial CMT (Mito-CMT)...
Research Opportunity: CMT-SORD
Research Opportunity: CMT-SORD I am writing to you today because there is a research opportunity...
2024 CMT Roadshow Coming to a City Near You?
Do you want to make a difference in CMT research? HNF is looking for patients with a confirmed CMT diagnosis to participate in the CMT Biobank
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.
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
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.
Cambridge-led natural history study identifies MTRFR/C12orf65 deficiency to improve diagnosis and therapy development
This research will potentially show a method that may be quicker and better at identifying...
Pharnext unveils the latest progress of the PREMIER Phase III clinical trial for CMT1A
The PREMIER trial, initiated in March 2021, is an international, randomized, double-blind, two-arm...
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