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.
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PFDD Meeting, September 28, 2018
On Friday, September 28, 2018 HNF hosted an Externally-led Patient Focused Drug Development (PFDD) Meeting. In attendance were patients, caregivers, Government Officials, Healthcare Providers, Industry Representatives, and Payors.
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).
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.
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