Seeking Participants for a New CMT Clinical Trial (for the US only): A Chance to Help Advance...
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TRIAD – Therapeutics
Total posts in Category: 4
Research Opportunity: CMT-SORD
Research Opportunity: CMT-SORD I am writing to you today because there is a research opportunity...
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.
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
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...
HNF is Reshaping Drug Repurposing for CMT
HNF has partnered with Rarebase, a public benefit precision medicine company that has screened a large library of FDA approved small molecules to identify candidates for various types of CMT. Their tech-enabled drug discovery platform is called Function™. There are many published discoveries on the genetic cause of many types of CMT, including an understanding of the basic mechanism of disease and potential targets for FDA-approved drug repurposing. It is this understanding that allows HNF and Rarebase to target the genetic root cause of CMT.
Can Existing Drugs Treat CMT?
HNF, in partnership with Rarebase, is leading the charge in the first-ever research initiative to tackle multiple types of CMT in one project using its tech-enabled drug discovery platform called “Function.”
SORD Webinar – Part 2 – Clinical Trial Update
Clinical Trial Update for SORD Deficiency – Applied Therapeutics – Dr. Shoshana Shendelman
SORD INSPIRE Phase III Clinical Trial
HNF Partner Applied Therapeutics is Currently Enrolling SORD Deficiency Patients for the INSPIRE Phase III Clinical Trial
CMT&ME App Study Presented at PNS Meeting 2022
The CMT&Me real-world digital lifestyle study self-reports data from patients with all types of CMT and is collected on a regular basis in both US and Europe.
Pharnext Announces On-Schedule Completion of Patient Enrollment in its Pivotal Phase III Trial of PXT3003, the PREMIER Trial, for the Treatment of Charcot-Marie-Tooth Disease Type 1A (CMT1A)
Pharnext Announces On-Schedule Completion of Patient Enrollment in its Pivotal Phase III Trial of PXT3003, the PREMIER Trial, for the Treatment of Charcot-Marie-Tooth Disease Type 1A (CMT1A)
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