NMD Pharma Receives FDA Clearance to Initiate Clinical Trial of NMD670 for CMT
NMD Pharma recently announced that it has received clearance from the US Food and Drug Administration (FDA) for its Investigational New Drug (IND) application to initiate a Phase 2 clinical trial of NMD670 in patients living with CMT types 1 and 2. NMD670 is a...
CMT Research Update/NMD Pharma
NMD Pharma recently published data demonstrating that their drug candidate, NMD670, improves muscle function and neuromuscular transmission deficits in both animal models and patients. Dr. William David Arnold, a member of the CMTRF Scientific Advisory Board, who was...Update from the Novartis CMT1A Development Team
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,...DTx Pharma Acquired by Novartis for CMT1A Therapy
Message From CMTRF CEO: This morning, DTx Pharma announced that it has been acquired by Novartis for an upfront payment of $500 million and additional payments of up to $500 million upon completion of pre-specified milestones. This is a very exciting next step in our...DTx Pharma Video – Using siRNA to Treat Charcot-Marie-Tooth Disease Type 1A
Important Research News Announcements
- Genomic Studies in Charcot-Marie-Tooth Disease July 31, 2024
- Charcot-Marie-tooth disease type 2A: An update on pathogenesis and therapeutic perspectives March 5, 2024
- Accelerate Clinical Trials in CMT (ACTCMT) Study March 1, 2024
- Creation of a Schwann Cell Gene Regulatory Network February 29, 2024
- Activation of XBP1s attenuates disease severity in models of proteotoxic Charcot-Marie-Tooth type 1B [Preprint] February 2, 2024
- The Regentime stem cell procedure, successful treatment for a Charcot-Marie-Tooth disease case December 28, 2023
- Preclinical Efficacy of Peripheral Nerve Regeneration by Schwann Cell-like Cells Differentiated from Human Tonsil-Derived Mesenchymal Stem Cells in C22 Mice December 18, 2023
- AAV-mediated editing of PMP22 rescues Charcot-Marie-Tooth disease type 1A features in patient-derived iPS Schwann cells November 28, 2023
- Combined RNA interference and gene replacement therapy targeting MFN2 as proof of principle for the treatment of Charcot-Marie-Tooth type 2A November 25, 2023
- Mouse models of human CNTNAP1-associated congenital hypomyelinating neuropathy and genetic restoration of murine neurological deficits October 19, 2023