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.


Learn more on this topic

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Sneak Peak of Fall Newsletter: CMT Research Study Survey

My name is Elizabeth Francisco and I am a graduate student from the Genetic Counseling program at the University of North Carolina Greensboro. I am inviting you to participate in a research study. The goal of my study is to learn more about the experiences of people with Charcot-Marie-Tooth (CMT) with genetic counseling and genetic testing. Adults with CMT and parents or legal guardians of someone of any age who has a diagnosis of CMT are eligible to participate

Building Awareness on CMT and Supporting the Patient Community – HNF Announces Support from Pharnext

HNFpharnext recently entered into a partnership with the French biopharmaceutical company, Pharnext, to help raise awareness of Charcot-Marie-Tooth (CMT) disease and support the CMT patient community through several initiatives. Building awareness is key! Pharnext’s support will assist HNF in distributing HNF’s CMT Update quarterly newsletter, enhancing the Global Registry for Inherited Neuropathies (GRIN), setting up activities for CMT September Awareness Month in the US and strengthening the CMT Inspire Community.

Quality of Life and CMT Research

Did you know that 95% of clinical trials fail? There are multiple causes, most related to efficacy or safety, which obviously can be harmful and risky for patients. The risk-reward of enrolling in trials is a judgment call based on the devastating effects of disease related to quality of life (QoL) or life-threatening disease. With CMT, the risk-reward is more of a challenging question for many, as CMT in most cases is non-fatal.

Help us answer questions that your doctors and the CMT Research Community aren’t too sure about.

Did you know that you can become part of a community in therapy development and further research for all forms of CMT and inherited neuropathies? The mission of the Global Registry for Inherited neuropathies (GRIN) is to collect clinical and genetic information from patients with ALL forms of Charcot-Marie-Tooth (CMT) and other related rare and ultra rare inherited neuropathies.

Hot off the press

It seems like almost weekly there is another new publication on CMT with interesting basic biology. While an earlier “Hot off the press” highlighted the work of Cherry and co-workers at the University of Texas Southwestern Medical Center who showed show that neurons lacking a gene for rab7 result in neuropathy.

New Study on CMT Type 2B

If we are to learn more about CMT and the effectiveness of rehabilitation it is worth asking the patient and their caregiver. A recent Italian study by Padua et al recently described a survey of CMT patients and caregivers and their perspectives and perceptions of rehabilitation efficacy and needs.

Allison Moore is going to be published!

HNF’s CSO (Chief Scientific Officer), Sean Ekins wrote a blog about his work with Allison Moore and her two “fighter Mom” friends Lori Sames and Jill Wood. He named his blog: “Rare disease heroes – Extraordinary collaborators we should be listening too.” Sean helped my friends and I write a paper called: “Multifaceted roles of ultra rare and rare disease patients/parents in drug discovery.” YES, Allison Moore is going to be published! It will be in Drug Discovery Today, soon. The link to the reprint is below.

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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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