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CMT-SORD: Share Your Story
…help accelerate advancements towards treatments and, ultimately, a cure for this disease. The main goal of this CMT research project is to understand better how CMT-SORD affects the person in…
CMTA Announces New Center of Excellence in Thailand
The Charcot-Marie-Tooth Association (CMTA), the world’s largest philanthropic funder of research aimed at bringing treatments to patients and improving the lives of those living with Charcot-Marie-Tooth disease (CMT), is excited…
CMTA’s $240K Investment Targets Gene Replacement Therapy in CMT4 Subtypes
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 $240K into a new gene replacement…
NMD Pharma Receives FDA Approval to Initiate Phase II Clinical Trial of NMD670 in CMT Patients
…clinical trial for their investigational drug, NMD670, in patients with Charcot-Marie-Tooth disease (CMT). This trial, named SYNAPSE-CMT, marks a significant milestone in the journey towards finding effective treatments for CMT….
CMTA Announces New Center of Excellence in the United Kingdom
The Charcot-Marie-Tooth Association (CMTA), the world’s largest philanthropic funder of Charcot-Marie-Tooth disease (CMT) research aimed at bringing treatments to patients and improving the lives of those living with Charcot-Marie-Tooth disease…
CMTA Alliance Partner, NMD Pharma, Announces Encouraging Data with Implications for CMT
…gravis and Charcot-Marie-Tooth disease this year.” “As we witness groundbreaking advancements in the treatment of myasthenia gravis, we’re filled with hope for the potential translational impact on CMT,” said Katherine…
Leading the Way: The CMTA Invests More Than $200K in Cutting-Edge CMT1A Organoid Research
The CMTA’s new $200K+ investment accelerates CMT1A organoid research, revolutionizing treatment evaluation for Charcot-Marie-Tooth disease (CMT) GLENOLDEN, PENNSYLVANIA, USA, February 21, 2024 — The Charcot-Marie-Tooth Association (CMTA), the largest philanthropic…
2023 CMTA-STAR Celebrating our Collective Achievements
…2023, and led to an increasing number of treatments heading to clinical trials. Notably, Novartis acquired DTx Pharma’s CMT1A treatment and will move it forward into the clinic. The CMTA’s…
Therapeutic Inactivation of CMT2 Disease Alleles with CRISPR
New technologies in gene editing such as CRISPR/Cas9 present a promising opportunity for the treatment of genetic diseases like CMT at the root cause – often just a single base…
Clinical Summit 2023 – Impact Report
…increasing levels of disability. CMT affects around 1 in every 2500 people, there are over 100 known causative gene mutations and there is currently no curative treatment available to the…