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Targeting Axon Degeneration – a treatment for all types of CMT?

Jan 7, 2020 | CMT Research Updates, Research news, Small Molecule Therapies, Therapy Types

Can axon degeneration be prevented in CMT? By Dr. Grace Pavlath, PhD I’m often asked if a therapy for one type of CMT could be beneficial in another. There are over 100 different genes known to cause CMT; they either directly disrupt myelin or axon integrity of...

AcuraStem Achieves First Milestone

Sep 18, 2019 | CMT Research Updates, Press Releases, Research news, Small Molecule Therapies, Uncategorized

Just nine months ago, we announced a milestone-based, research collaboration with AcuraStem to test thousands of compounds aimed at producing effective treatments for CMT2A. We are very pleased to report that AcuraStem has completed phase one of the CMT2A project and...

CMT1B project is funded & ready for take off!

Aug 2, 2019 | CMT Research Updates, Press Releases, Research news, Small Molecule Therapies

Paving pathways for mission critical projects CMT Research Foundation is partnering with Dr. Maurizio D’Antonio of I.R.C.C.S. Ospedale San Raffaele, in Milan, Italy to fund the lab’s research of Myelin Protein Zero (MPZ), the gene associated with CMT1B.  Dr....

CMTRF Responds to FDA Fast Track Designation of PXT3003

Feb 11, 2019 | CMT Research Updates, Research news, Small Molecule Therapies

The FDA Grants Fast Track Designation to Pharnext’s PXT3003 for CMT1A Pharnext, an advanced clinical-stage biopharmaceutical company, created a drug combination of three drugs: baclofen, naltrexone and sorbitol known as PXT3003. Baclofen is known to relieve...

CMTRF Inks Milestone-Driven Partnership with Acurastem to Repurpose Established Drugs for the Treatment of CMT

Dec 20, 2018 | Press Releases, Research news, Small Molecule Therapies, Uncategorized

Focus will be on expansion of AcuraStem’s patient-based discovery platform, iNeuroRx™, to leverage patient neurons and advanced informatics technology to identify promising drugs ATLANTA (December 20, 2018)The newly launched CMT Research Foundation (CMTRF), a...

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RSS Important Research News Announcements

  • Natural history of Charcot-Marie-Tooth disease type 2A: a large international multicentre study January 9, 2021
  • Epigenetic Regulation of CMT: A Lesson from Drosophila Models January 7, 2021
  • Clinical features of homozygous FIG4-p.Ile41Thr Charcot-Marie-Tooth 4J patients January 6, 2021
  • Aberrant Splicing in GJB1 and the Relevance of 5′ UTR in CMTX1 Pathogenesis December 28, 2020
  • Plasma neurofilament light chain as a potential biomarker in Charcot-Marie-Tooth disease December 20, 2020
  • Neurogene adds another $115M to the pot for trials for its gene therapies, including its CMT4J program December 18, 2020
  • More Than Hope: An Action Plan to End CMT December 18, 2020
  • Aminoacyl-tRNA synthetases in Charcot-Marie-Tooth disease: a gain or a loss? November 26, 2020
  • The expanding genetic landscape of hereditary motor neuropathies November 19, 2020
  • Phase I/IIa Trial of scAAV1.tMCK.NTF3 for Treatment of Charcot-Marie-Tooth Type 1A (CMT1A) November 19, 2020
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