Two recent publications from Pharnext describe a novel synergistic combination of 3 drugs (baclofen, naltrexone and sorbitol) and its effect on CMT1A both in the lab and in a phase II clinical trial. These 3 drugs already approved but for unrelated conditions, are combined at new optimal lower doses and under a new formulation. This novel potential therapeutic is called PXT-3003.
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Support CMT Therapeutic Alliance
HNF has entered into a joint venture – the CMT Therapeutic Alliance – with a unique non profit organization (BioPontis Alliance for Rare Diseases) that brings professional drug discovery capabilities to translate our research results into potential treatments.
Scientific Advisory Board Meeting
On the 7th of November we convened our scientific advisory board meeting at the HNF offices in NY. We have written a detailed review that has been published and captures all of the discussion and make this freely available to the scientific community.
Finding Experts Health Care Providers for Charcot-Marie-Tooth
We are creating an innovative HCP Directory which provides these clinicians with additional tools to care for their patients.
Breaking News: First Therapeutic Gene Therapy to Treat an Inherited Neuropathy is Approved for Clinical Trial!
The first disease community to receive a therapeutic gene to the spinal cord for an ultra rare inherited neuropathy is Giant Axonal Neuropathy (GAN). Congratulations to Hannah’s Hope Fund (HHF), a 501(c)3 public charity, which has driven this collaborative research in less than six years. Six million dollars has been raised to date to fund pre-clinical and clinical research on this rare disease.
Saturday April 18, 2015: Jillian’s Cure SUPERHERO 5K
Jillian’s Cure SUPERHERO 5K
Saturday, April 18, 2015
at 7:00am
111 Maiden Lane
Lexington, SC 29072
Sunday May 1, 2016: 2016 TD Bank Five Boro Bike Tour
HNF is thrilled to have been chosen for the fifth year as a Charity Partner for the 2016 TD Bank Five Boro Bike Tour.
Saturday July 25, 2015: The Rochester Chrome Divas Charity Benefit
To raise awareness and fund research for CMT6 in Webster, New York!
August 16, 2015 Grace’s Courage Crusade Eighth Annual Fundraiser
Please join us for this amazing event to benefit Grace’s Courage Crusade, a passionate mission of the Sidoti/Caldarone family to support those living with Charcot-Marie-Tooth disease
Pharnext Announces Pleotherapy Proof of Concept in Charcot-Marie-Tooth Disease Type 1A
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 Rare Disease Patient That Does It All!
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.
Hot Off the Press
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.
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