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.
A movie script could not play out like this. There are very few occasions when a rare disease patient becomes the topic of a story that is truly uplifting. Often the stories are tragic or disheartening. That was not the case, however, with CMT patient Kim Goodsell.
One of the ways that some CMT patients first become aware of their disease is when they are given a drug treatment for another disease. This is termed chemotherapy-induced neurotoxicity. Drugs such as paclitaxel and the vinca alkaloids that are widely used in cancer treatment cause severe peripheral neuropathy and in some patients this exacerbates CMT, revealing it perhaps for the first time.
My name is Courtney Hollett, Fundraising Coordinator at the Hereditary Neuropathy Foundation. This time of year I count my blessing daily and wanted to share with you a Q&A session I had with a new supporter of HNF. Kara, like myself has many family members affected with CMT and I reached out to her to share her thoughts and advice about how to “support” a friend or family member that’s affected with Charcot-MarieTooth.
Hereditary Neuropathy Foundation (HNF) is pleased to announce the creation of a joint venture to develop drug candidates for the treatment of Charcot-MarieTooth (CMT) Disease with BioPontis Alliance for Rare Disease.
In June 2013, HNF made the important decision to refine the organization’s mission. We now place more importance on new initiatives that take our research discoveries and translate them to the next phase in the drug discovery process.
We divide our research initiatives into 3 areas:
We at HNF are so proud of the accomplishments of those scientists we fund and are asking you to please continue to support our efforts. Our Therapeutic Research In Accelerated Discovery (TRIAD) program is a proven collaborative model in the drug discovery process.
Grace’s Courage Crusade, a passionate campaign of the Sidoti/Caldarone family to raise awareness and fund CMT research, held its annual “Brunch by the Beach” fundraiser in scenic Newport, RI on November 2, 2014.
Morgan Johnson doesn’t have Charcot-Marie-Tooth, but she is quickly learning everything she can about it. Born and raised near the coast in Galveston, Texas, Morgan, 26, has been a competitive runner and swimmer since she was little. Four years ago, she began participating in triathlons. In 2009, as an undergrad at the University of North Texas, Morgan and her partner, Sean Thompson started a Youth Triathlon Team for 7-14 year olds. Known as the North TX Tri Team, they have remained together and still compete.
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
FACEBOOK CHAT: “Rare Neuropathies: Getting Diagnosed, Getting Help”
WHEN: September 17, 2014 (7-8:30 p.m. EST)
WHERE: www.facebook.com/NeuropathyAssociation
Occupational Therapy can provide innovative, individualized approaches for your CMT symptoms.
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