CATEGORIES: CMT Type - CMT1A | CMT Type - CMT1B | CMT Type - CMT1X | CMT Type - CMT2A | CMT Type - CMT2C | CMT Type - CMT4A GDAP1 | CMT Type - CNTNAP1 | CMT Type - GAN | CMT Type - HDAC6 | CMT Type - HNPP | CMT Type - MTRFR-C12orf65 | CMT Type - SORD Deficiency | Research | Types of CMT | Upcoming Events

2024 CMT Roadshow Coming to a City Near You?

by | Mar 26, 2024 | 0 comments

Click to Participate

The CMT Biobank is now open! Are we coming to a city near you?

Do you want to make a difference in CMT research? HNF is looking for patients with a confirmed CMT diagnosis to participate in the CMT Biobank. Your vital blood samples and de-identified GRIN data will be shared to develop cell models of all CMT subtypes, as targets for various therapeutic approaches, and for the identification of biomarkers of CMT.

It’s easy. Simply complete the form if you are able to visit one of the following events where samples will be collected by our partner, COMBINEDBrain. There is no cost to participate.

2024 Dates:

April 4th-5th – Orlando, FL

June 7th-8th – San Diego, CA

July 20th-21st – Philadelphia, PA

July 27th-28th – Philadelphia, PA

July 27th-28th – Seattle, WA

September 27th-28th – Atlanta, GA

October 19th – Nashville, TN

December 4th-5th – Los Angeles, CA

Click to Participate

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Hot Off the Press – Potential Treatment for CMT1A

by | Mar 9, 2015 | , , | 53 Comments

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.

Support CMT Therapeutic Alliance

by | Mar 9, 2015 | , | 0 Comments

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.

Breaking News: First Therapeutic Gene Therapy to Treat an Inherited Neuropathy is Approved for Clinical Trial!

by | Mar 6, 2015 | , , | 9 Comments

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.

Pharnext Announces Pleotherapy Proof of Concept in Charcot-Marie-Tooth Disease Type 1A

Pharnext Announces Pleotherapy Proof of Concept in Charcot-Marie-Tooth Disease Type 1A

by | Dec 19, 2014 | , , | 12 Comments

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.

Sneak Peak of Fall Newsletter: CMT Research Study Survey

by | Sep 16, 2014 | , | 2 Comments

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

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.

Targeting PMP22 in CMT1A Patients with Gene Duplication

by | Aug 19, 2014 | , | 1 Comment

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.

Quality of Life and CMT Research

by | Jul 7, 2014 | | 4 Comments

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.

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