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

by | Aug 19, 2014 | 1 comment

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.  Pharnext is an advanced clinical stage biopharmaceutical company discovering and developing new therapeutics that target multiple key disease pathways for orphan and common neurological diseases including Charcot-Marie Tooth disease. The company’s lead program PXT-3003 for the treatment of CMT 1A has completed Phase 2 clinical trials and pending discussions with the appropriate regulatory agencies will undergo Phase 3 clinical trials. PXT-3003 was recently granted Orphan Drug designation from the European Medicines Agency and the U.S. Food and Drug Administration. Learn more at www.pharnext.com or www.hnf-cure.org.

research progress

Learn more on this topic

Related Blog Posts

In Memoriam

The HNF family has lost two great people this summer: Franklin G. Downing Sr. (father of board member Matthew Downing) and George G. Gatta (father of board member Kathleen Zappola).

Targeting PMP22 in CMT1A Patients with Gene Duplication

In previous collaborative work with a group of investigators including Dr Rolf Renne from the University of Florida, Dr. Alex Murashov from East Carolina University and Dr. Lynn Hudson from the NIH-NINDS, we validated a microRNA known as miR29a as a reagent that corrected the expression level of PMP22 in rodent Schwann cells.

September is CMT Awareness Month

CMT awareness month is an entire month dedicated to building awareness, raising funds and finding a cure. The Hereditary Neuropathy Foundation (HNF) has committed to spend 30 days in September hosting local and national events, launching fundraisers, and spreading the word about the effects of Charcot-Marie-Tooth disease

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.

Join the conversation

Leave a Comment

1 Comment

  1. Heidi Kirschner

    Millions suffer from acute or chronic pain every year and the effects of pain exact a tremendous cost on our country in health care costs, rehabilitation and lost worker productivity, as well as the emotional and financial burden it places on patients and their families. While acute pain is a normal sensation triggered in the nervous system to alert you to possible injury and the need to take care of yourself, chronic pain is different. Chronic pain persists. Pain signals keep firing in the nervous system for weeks, months, even years. Pain affects more Americans than diabetes, heart disease and cancer combined. According to the Institute of Medicine of The National Academies chronic pain affects 100 million Americans. Nyloxin provides all-natural anti-inflammatory analgesic pain relief that is non-toxic, non-narcotic, non-addictive, non-steroidal and contains no aspirin or acetaminophen. Safe to use without a prescription, it treats conditions that cause chronic pain. It’s the only thing that has helped with my CMT and with my Occipital Neuralgia.Help those that are suffering and get paid to do so or use as a means of fundraising for more research!

    Reply

Submit a Comment

Your email address will not be published. Required fields are marked *

Newsletter

Join for notifications on events, campaigns, & news