pharnext news

Exciting News For The CMT Patient Community

by | Dec 20, 2018 | 2 comments



HNF – Pharnext collaborative highlights:  

  • Collaborative relationship since 2013.
  • Identified the HNF Center of Excellence clinical sites for the Adult Phase III clinical trial for PXT3003 and HNF was essential in patient recruitment.
  • Created awareness and expanded knowledge in the medical professional communities on CMT (coordinated important conferences, dissemination of educational materials, and media outreach).

October 16, 2018: Pharnext Announced Positive Topline Results from Pivotal Phase 3 Trial of PXT3003 for CMT1A  

  • HNF is instrumental in setting up the pediatric clinical trial sites for PXT3003 (launch mid 2019).  
  • HNF’s Externally-led PFDD Meeting for FDA helps inform and raise critical awareness on the impact CMT has on a patient’s quality of life.  
  • HNF supports the CMT&ME App (sponsored by Pharnext), an international observational realworld data study to explore the impact of CMT on patients in the real-world setting.

“Thank you to the Hereditary Neuropathy Foundation and their Centers of Excellence for assisting Pharnext with patient recruitment for PXT3003. We are thrilled with the outcome of the trial and with the clearly demonstrated efficacy of PXT3003 in addressing the debilitating disease progression of CMT1A.” – Professor Daniel Cohen, MD, PhD, Pharnext’s Co-Founder and Chief Executive Officer.


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  1. Teresa M Carroll

    What is the application of this treatment for CMT1X?

  2. Rich carlson

    I’m an identical twin and my brother and I have CMT1a and we both have one child with CMT1a and we’re wondering how we can get started with this treatment for us and or maybe our children.


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