Research News and Updates
Read the latest from the CMT Research Foundation on drug development and clinical advances.
Continuation of Inflammation Research for CMT1B
By: George Simpson In 2020, the CMT Research Foundation had funded renowned nerve disease expert, Dr. Rudolf Martini of University Hospital Würzburg (Germany), to investigate whether inhibiting inflammation in the peripheral nerves could reduce symptoms and improve...
Optimal Dosing Identified in CMT1X Research Update
By: Kelsie Timbie A CMT Research Foundation-funded project at the University of Illinois Chicago has identified optimal dosing for a potential therapeutic for CMT1X. Like other forms of CMT, cell-to-cell communication within the peripheral nervous system malfunctions...
CMT2E Gene Therapy Project Successfully Completes Milestone 2
By: Kelsie Timbie, CMT Research Foundation A collaboration between Dr. Chris Lorson and Dr. Michael Garcia of the University of Missouri and funded by the CMT Research Foundation is producing promising results in an innovative new approach to treating CMT2E. CMT2E...
Italian Research Group Completes CMT Research Foundation-Funded Project, Results Encourage Further Work in New Therapy for Adult and Elderly Patients with CMT1A/CMT1B
By: Kelsie Timbie, CMT Research Foundation The CMT Research Foundation is pleased to announce that Dr. Maurizio D'Antonio's research team at San Raffaele Scientific Institute in Milan, Italy has completed their CMT Research Foundation-funded project investigating the...
2022 at CMTRF
Dear Friends of CMTRF, In preparing for another busy year at CMTRF, I took some time recently to look back at the accomplishments of 2022. I am immensely proud of what our team has achieved (summarized below) and I believe it underscores how CMTRF adds significant...
Muscular Dystrophy Association and CMT Research Foundation Announce Joint Research Study Grant
(New York) December 14, 2022 The Muscular Dystrophy Association (MDA) and CMT Research Foundation (CMTRF) announce joint funding for a study by Alessandra Bolino, PhD, at San Raffaele Hospital (Ospedale San Raffaele), Italy, on restoring membrane trafficking in...
Dr. Arthur Suckow, DTx Pharma Inc. – FALCON
One of the first research teams the CMT Research Foundation (CMTRF) funded was DTx Pharma, Inc., and the project focused on delivery of genetic medicine to the myelinating Schwann cells of the peripheral nerves - a major scientific challenge to effectively treating...
CMT Research Foundation partners with Samsara Therapeutics to develop a novel class of drugs in CMT1A
By: Keith Fargo, PhD, Chief Science Officer The CMT Research Foundation is pleased to announce a new project with Samsara Therapeutics, a US/UK-based biotech company that is developing a novel class of drugs for CMT1A. CMT1A is caused by duplication of a stretch of...
CMT Research Foundation and Samsara Therapeutics Partner on a Novel Therapeutic Approach for CMT1A with the Potential to Reach Clinical Trials
ATLANTA (October 13, 2022) The CMT Research Foundation (CMTRF), a non-profit focused solely on delivering treatments and cures for Charcot-Marie-Tooth disease*, has invested in Samsara Therapeutics Inc., a biotech company focusing on discovering and developing...
CMT Research Foundation Awards $152,524 to ORYZON to test novel HDAC6 inhibitors in CMT1A
By: Keith Fargo, PhD, Chief Science Officer New project will examine impact of two selective and potent drug candidates recently discovered by ORYZON The CMT Research Foundation is delighted to announce a new project with ORYZON, a clinical stage pharmaceutical...
Important Research News Announcements
- 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
- Intravenous Administration of an AAV9 Vector Ubiquitously Expressing C1orf194 Gene Improved CMT-Like Neuropathy in C1orf194-/- Mice October 16, 2023
- Clinical trials in Charcot-Marie-Tooth disorders: a retrospective and preclinical assessment September 22, 2023
- Structures of wild-type and selected CMT1X mutant connexin 32 gap junction channels and hemichannels August 30, 2023
- AAV9-mediated SH3TC2 gene replacement therapy targeted to Schwann cells for the treatment of CMT4C August 28, 2023
- Proteostasis plays an important role in demyelinating Charcot Marie Tooth disease August 19, 2023
- Gene replacement therapy in two Golgi-retained CMT1X mutants before and after the onset of demyelinating neuropathy August 2, 2023
- Amlexanox: Readthrough Induction and Nonsense-Mediated mRNA Decay Inhibition in a Charcot-Marie-Tooth Model of hiPSCs-Derived Neuronal Cells Harboring a Nonsense Mutation in GDAP1 Gene July 24, 2023
- Monoallelic variation in DHX9, the gene encoding the DExH-box helicase DHX9, underlies neurodevelopment disorders and Charcot-Marie-Tooth disease July 18, 2023
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