News & Stories

See the latest news about CMT drug development and read stories from the CMT community that highlight why we must deliver treatments and cures during our lifetime.

Vanderbilt’s Charles Sanders Successfully Finds Molecules That Alter PMP22 Production or Cell Surface Trafficking; In Next Phase Will Test if They Can Improve CMT-like Problems in Schwann Cells

Mar 6, 2024 | CMT Research Updates, CMTRF Funded Research

CMT1A is caused by a gene-copying event that results in the overproduction of the peripheral myelin protein 22 (PMP22) in Schwann cells. The excess PMP22 protein fails to traffic normally to the cell surface and instead collects inside the cells as clumps of proteins...

CMT Research Foundation Partners to Advance Study of CMT1J by Dr. Stephan Zuchner

Feb 20, 2024 | CMT Research Updates, CMTRF Funded Research

The CMT Research Foundation, a non-profit focused solely on delivering treatments and cures for Charcot-Marie-Tooth disease (CMT), has partnered with the 1J Foundation, a 501(c)(3) organization dedicated to finding a cure for patients with 1J, a newly identified...

Project Update: Nanite Looks to Advance Polymer-Based Encapsulation for CMT1A

Feb 9, 2024 | CMT Research Updates, CMTRF Funded Research

CMT 1A results from the duplication of the myelin protein 22 gene (PMP22) in Schwann cells. This causes excessive production of the PMP22 protein which disrupts the myelination process of peripheral nerves and ultimately results in axonal loss and muscle wasting over...

Update from the Novartis CMT1A Development Team

Oct 29, 2023 | About CMT, Drug Development

As you know, the CMT Research Foundation is proud to have introduced DTx leadership to Charcot-Marie-Tooth disease in 2019. We funded their first work in CMT to demonstrate the FALCON platform's potential to deliver a drug to the Schwann cells. And from there, the...

ARM-101: A Potential “Precision Drug Therapy” For CMT1A

Oct 24, 2023 | CMTRF Funded Research, Research News

The CMT Research Foundation has invested in a project at Armatus Bio, Inc. that will advance a novel miRNA gene therapy candidate for CMT type 1A. Principal Investigator Dr. Brian Price and his colleagues aim to harness the potential of microRNAs (miRNAs), snippets of...

CMT Research Foundation Invests in Armatus Bio, Inc. to Advance Potential Therapeutic for CMT1A

Oct 23, 2023 | CMT Research Updates, CMTRF Funded Research

ATLANTA (October 24, 2023) The CMT Research Foundation, a non-profit focused solely on delivering treatments and cures for Charcot-Marie-Tooth disease (CMT)*, has invested in a project at Armatus Bio that will advance a novel miRNA gene therapy candidate for CMT type...

Dr. Afrooz Rashnonejad Completes Initial Studies Demonstrating the Effectiveness of a Novel Gene Therapy for CMT1B

Sep 30, 2023 | CMTRF Funded Research

Charcot-Marie-Tooth type 1B is caused by mutations myelin protein zero, which is an essential component making myelin, the protective layer or sheath around nerves. Defective myelin protein zero results in damaged or impaired myelin leading to progressive nerve damage...

Another Asset with Potential to Treat Charcot-Marie-Tooth has been Acquired by Pharma

Sep 26, 2023 | CMT Research Updates, CMTRF Funded Research, Drug Development, Research News

An asset that is being tested in CMT4B1 underwritten by the CMT Research Foundation has been licensed by AcuraStem to Takeda for approximately $580 million. The exclusive, worldwide license will enable Takeda to develop and commercialize AcuraStem's PIKFYVE targeted...

Endgame Campaign for CMT1A Tops $6.6 Million

Sep 20, 2023 | CMT Research Updates, CMTRF Funded Research

Recently, Peter J. deSilva, CMTRF Board member and Chair of the ENDGAME Capital Campaign to end CMT1A and Susan Ruediger, CMT patient, co-founder, and Chief Mission Officer of the CMTRF, hosted a private call with donors to update them on the progress being made in...

Oryzon Develops Novel HDAC6 Inhibitor ORY-4001 Showing Therapeutic Potential for CMT in Mouse Model

Jul 18, 2023 | CMT Research Updates, CMTRF Funded Research, Research News

Scientists have been studying a potential treatment option for CMT by inhibiting (blocking) a protein called histone deacetylase 6 (HDAC6). They have found that HDAC6 plays an important role in regulating the pathways involved in breaking down proteins and...