CMT News & Updates
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.
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Project Update: Nanite Looks to Advance Polymer-Based Encapsulation for CMT1A
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…
Read More : Project Update: Nanite Looks to Advance Polymer-Based Encapsulation for CMT1A
ARM-101: A Potential “Precision Drug Therapy” For CMT1A
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…
Read More : ARM-101: A Potential “Precision Drug Therapy” For CMT1A
CMT Research Foundation Invests in Armatus Bio, Inc. to Advance Potential Therapeutic for CMT1A
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…
Dr. Afrooz Rashnonejad Completes Initial Studies Demonstrating the Effectiveness of a Novel Gene Therapy for CMT1B
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…
Another Asset with Potential to Treat Charcot-Marie-Tooth has been Acquired by Pharma
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…
Read More : Another Asset with Potential to Treat Charcot-Marie-Tooth has been Acquired by Pharma
Endgame Campaign for CMT1A Tops $6.6 Million
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,…
Oryzon Develops Novel HDAC6 Inhibitor ORY-4001 Showing Therapeutic Potential for CMT in Mouse Model
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…
DTx Pharma Acquired by Novartis for CMT1A Therapy
Novartis acquires DTx's FALCON technology to advance treatment development for CMT1A
Read More : DTx Pharma Acquired by Novartis for CMT1A Therapy
DTx Pharma
We are pleased share this story that published on May 3: https://patientworthy.com/2023/05/03/dtx-pharma-developing-cmt1a-rna-therapeutics/ One of the early programs CMTRF funded was DTx, a young company that had developed a new platform for…
