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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Clinical Trial for CMT1A begins in Korea
Helixmith, specializing in gene therapy research for over 20 years, has kicked off its phase I and 2a clinical trial for using VM202 (Engensis) to treat CMT1A.
Press Release: Pharnext provides regulatory and clinical update on PXT3003 Phase III study for the treatment of Charcot-Marie-Tooth Type 1A
US Food and Drug Administration has agreed with Pharnext and provided clear guidance on the regulatory pathway to approval for PXT3003, including key design elements of a single pivotal Phase III study
ACE-083 did not achieve statistically significant improvements in functional endpoints relative to placebo; Acceleron to discontinue development of ACE-083.
treatment with ACE-083 in patients with Charcot-Marie-Tooth disease (CMT) did not demonstrate functional improvement in the Phase 2 trial
PFDD Meeting Sessions
Welcome and Opening Remarks Overview: What is CMT? - Michael Shy, MD Overview: What is CMT? - Dr....
The Externally-led Patient-Focused Drug Development (PFDD) Meeting Was A Huge Success!
HNF successfully executed the Externally-led PFDD Meeting for the FDA on September 28th, 2018.
HNF’s Landmark Externally-Led Patient-Focused Drug Development Meeting
HNF will be holding a landmark externally-led Patient-Focused Drug Development Meeting for CMT and INs in Washington, D.C. on Friday, September 28, 2018.
HNF’s Roadmap To Clinical Trial Development
2018 will be “the year” for our community as we prepare for the externally-led Patient-Focused Drug Development Meeting.
Landmark “Voice of the Patient” PFDD CMT Meeting Needs All Hands on Deck!
Help support this ground-breaking PFDD CMT Meeting being held on September 28, 2018 in Washington, D.C.
Hope on the Horizon: CMT & FDA Patient-Focused Drug Development Meeting for Charcot-Marie-Tooth to be Held in 2018
HNF will be holding a landmark externally-led Patient-Focused Drug Development (PFDD) Meeting CMT & INs in Washington, D.C. on September 28, 2018. The meeting will be held in conjunction with September National CMT Awareness Month.
Pharnext’s lead PLEODRUG © for treatment of CMT1
Pharnext completed patient enrollment for the international Phase 3 clinical trial of PXT3003, Pharnext’s lead PLEODRUG ©, for treatment of CMT1A.
Are You a Patient With CMT 1A? New Clinical Trial Opportunity Available For You!
For the first time investigators are looking for patients to participate in a pivotal Phase 3 clinical trial of Pharnext’s lead investigational pleodrug, PXT-3003 for the potential treatment of Charcot-Marie-Tooth Disease Type 1A (CMT1A).
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