Charcot Marie Tooth Association News
Charcot Marie Tooth Association News
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Find the latest news regarding our research and events. Founded by patients in 1983, the vision of the CMTA has always been a "A World Without CMT" and we will accomplish this mission by accelerating research and empowering patients.
Charcot-Marie-Tooth Association (CMTA) Alliance Partner, NMD Pharma, announced the publication of comprehensive data sets demonstrating for the first time the ability of CIC-1 inhibition to restore muscle function in patients suffering from neuromuscular junction (NMJ) transmission deficits. These data are ...
Charcot Marie Tooth Association News
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2w ago
CMTA Alliance Partner, NMD Pharma, Announces Encouraging Data with Implications for CMT ..read more
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The Charcot-Marie-Tooth Association (CMTA), the largest philanthropic funder of Charcot-Marie-Tooth disease (CMT) research aimed at bringing treatments and a cure to patients, is pleased to announce the appointment of Bernard Coulie, M.D., Ph.D., and Kevin Marks to its Board of ...
Charcot Marie Tooth Association News
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2w ago
Evolution: CMTA Board Welcomes Coulie & Marks ..read more
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The Charcot-Marie-Tooth Association (CMTA), the largest philanthropic funder of Charcot-Marie-Tooth (CMT) disease research aimed at bringing treatments to patients, announced today an investment of $450K into a new scientific approach for CMT1A and CMT1B, two common forms of the disease. ...
Charcot Marie Tooth Association News
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2w ago
CMTA’s $450K Investment Targets New Approach to Treatment for Patients with CMT1A and CMT1B ..read more
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It is with the heaviest of hearts that the Charcot-Marie-Tooth Association (CMTA) mourns the loss of our friend and Strategy to Accelerate Research (STAR) Scientific Advisory Board (SAB) Member, Dr. M. Laura Feltri. Dr. Feltri passed away December 25, 2023, ...
Charcot Marie Tooth Association News
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1M ago
The CMTA Mourns the Loss of Scientific Advisory Board Member and Esteemed CMT Clinical Researcher ..read more
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The Charcot-Marie-Tooth Association (CMTA), the largest philanthropic funder of Charcot-Marie-Tooth disease (CMT) research that’s aimed at bringing treatments and a cure to patients of this rare disease, is pleased to announce the appointment of Dr. Charlotte Sumner to its Strategy ...
Charcot Marie Tooth Association News
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2M ago
The CMTA Appoints Professor Charlotte Sumner, M.D., to its Scientific Advisory Board ..read more
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In a statement released on February 15, 2024, by Charcot-Marie-Tooth Association (CMTA) Alliance Partner, Applied Therapeutics, the company said that its INSPIRE Phase III trial for a certain type of Charcot-Marie-Tooth disease (CMT), called SORD-deficiency, showed positive interim 12-month results ...
Charcot Marie Tooth Association News
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2M ago
CMTA Alliance Partner, Applied Therapeutics, Announces Positive 12-Month Results in its Ongoing INSPIRE Phase III Trial for SORD-CMT ..read more
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Multisite and multiyear collaborative project led by a team of international researchers Advancing CMT Treatment with CRISPR-Cas9 and small molecule drug development GLENOLDEN, PA, February 7, 2024 – The Charcot-Marie-Tooth Association (CMTA) announced today a groundbreaking international research initiative investing over ...
Charcot Marie Tooth Association News
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2M ago
CMTA Awards Over Half a Million Dollars to Fuel Research in Demyelinating Forms of CMT ..read more
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Significant Research Investment Made in 2023: We are excited to share that in 2023, the CMTA has ongoing and committed investments of $5,000,00 in research dedicated to finding effective treatments and ultimately a cure for Charcot-Marie-Tooth disease. Support for Natural History Studies Drives ...
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3M ago
2023 CMTA-STAR Celebrating our Collective Achievements ..read more
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CMTA Alliance Partner ToolGen announced December 18, 2023 that it received Orphan Drug Designation (ODD) for its rare disease gene correction treatment, TGT-001, from the U.S. Food and Drug Administration (FDA). TGT-001 targets Charcot-Marie-Tooth (CMT), a rare genetic disease related ...
Charcot Marie Tooth Association News
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4M ago
ToolGen CMT1A Treatment Receives Orphan Drug Designation ..read more
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French biopharmaceutical company Pharnext SA announced Dec. 11 that the initial results from its Phase III clinical trial (PREMIER trial) for CMT1A did not meet expectations. The company said in a news release that analyzing the results was challenging because of ...
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4M ago
Pharnext Announces Disappointing Results for CMT1A Drug Trial PXT300 ..read more
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