PARIS, December 18th, 2014 – Pharnext SAS today announced the proof of concept of its pleotherapy research and development approach based on a proprietary network pharmacology platform that identifies synergic combinations of drugs already approved for other diseases. Indeed, Pharnext’s lead pleodrug, PXT-3003, has shown positive results both in preclinical and Phase 2 clinical studies published today in the Orphanet Journal of Rare Diseases.
The Hereditary Neuropathy Foundation and BioPontis Alliance for Rare Disease Partner to Develop Treatments for Charcot-Marie-Tooth
BioPontis Alliance for Rare Disease and the Hereditary Neuropathy Foundation (HNF), both philanthropies, announced today the creation of a joint venture to develop drug candidates for the treatment of the rare disease known as Charcot- Marie-Tooth (CMT) Disease.
BioPontis Alliance announced its alliance model earlier this month. Today’s partnership announcement with HNF is a first demonstration of a collaborative model where researchers and all CMT dedicated organizations can come together for a common cause – treatments.
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.
My daughter Dakota is a force to be reckoned with, a strong willed, happy, smart young lady with a wit that could knock you off your chair with laughter. She’s had the personality of a leader since her very first day on the Kindergarten soccer field. Never one to be a wall flower but beautiful as a rose. The light she has carried around since birth is contagious and could brighten almost any situation.
That light dimmed a bit when, at age eleven, Dakota was diagnosed with CMT1A. We hadn’t the slightest idea what CMT was or where it had come from. It turned out that after some testing, we learned that my husband, Dakota’s father, has a mild case of CMT1A that was never diagnosed.
“FAITH, LOVE AND HOPE KEEPS TIMMY DIXON GOING!”
I often wonder what it’s like to be like everyone else out there. How great it would be to do things most consider easily accessible and within reach. I believe in myself; it’s not really about that. Sometimes I just feel alone, cold, and bitter after considering life in general. Then I ponder a thought: there are too many hurdles I’ve already jumped over
Research on CMT is global, and covers both laboratory and clinical studies. It is critically important to be aware of what is happening elsewhere as well as in the USA because it can have implications for what we do and fund at the HNF.
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.
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.
A bright, crisp day greeted supporters of Grace’s Courage Crusade as they gathered to enjoy brunch by the beach with breathtaking views at the Atlantic Beach Club in Newport, RI. Following the amazing buffet spread, the crowd got intense over a dessert auction that offered tempting sweets and goodies provided by local businesses.
We are delighted to announce that after volunteering for HNF for the past year Dr. Sean Ekins, Ph.D, D.Sc. will officially become our CSO responsible for overseeing the TRIAD program, directing our scientific advisory board, and communicating our research initiatives to the scientific community. In addition he serves as the Principal Investigator of the clinical Global Registry for Inherited Neuropathy (GRIN).