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….
Hell’s Kitchen Broadway Show
09/28/2024 @ 2:00 pm – 5:00 pm – Hell’s Kitchen is a new musical created by 15-time Grammy Award winner Alicia Keys. Featuring her new original songs and iconic anthems in a story inspired by her own New York experience, this is the kind of Broadway musical of which dreams are made. Hell’s Kitchen has been nominated for thirteen Tony Awards including Best […]
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…
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…
Nanoparticles for CMT1A, CMT1B, and CMTX1: A New Frontier in Gene Therapy
…cells while remaining capable of delivering the necessary gene therapies effectively. This delicate balance is crucial for the success of the treatment, as the nanoparticles must be able to traverse…
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…
Help the CMTA Develop Clinical Trials in a Dish
…that are affected by CMT disease. If we can study these cells, we can increase our understanding of CMT and test the effectiveness of new treatments more quickly. Stem cells…