CATEGORIES: Clinical Trials | CMT Type - CMT1A | Industry | Pharnext | Research | Research and Critical Trials | TRIAD
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Great News From HNF TRIAD Industry Partner — Pharnext

by | Aug 2, 2023 | 0 comments

Pharnext, a biopharmaceutical company, has announced new positive results from the ongoing open-label extension study of their drug PXT3003 in Charcot-Marie-Tooth Disease Type 1A (CMT1A), called the PLEO-CMT-FU trial. The trial has been conducted for a total of 6 years, and the data is consistent with a long-term treatment benefit of PXT3003 in patients with mild-to-moderate CMT1A. The results indicate that PXT3003 has a good safety profile and shows a long-term treatment effect on the Overall Neuropathy Limitation Scale (ONLS), which measures functional motor disability. A group of 117 patients with mild-to-moderate CMT1A are still receiving treatment with the high dose of PXT3003 in the trial.

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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.

Help us answer questions that your doctors and the CMT Research Community aren’t too sure about.

by | May 19, 2014 | | 0 Comments

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.

Hot off the press

by | Feb 28, 2014 | | 0 Comments

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.

New Study on CMT Type 2B

by | Feb 12, 2014 | | 5 Comments

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.

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