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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by | May 27, 2015 | , , | 0 Comments

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

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by | Mar 9, 2015 | , | 0 Comments

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

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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.

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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.

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