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Exploring New Therapeutic Pathways for CMT1B: UPR Activation
…developing a potential treatment for CMT1B. This groundbreaking work, led by Maurizio D’Antonio, PhD, focuses on activating the Unfolded Protein Response (UPR), targeting the fundamental issue caused by mutations in…
Central Tennessee CMTA Branch Meeting (Virtual)
08/26/2024 @ 6:00 pm – 7:00 pm – Please join us (via Zoom) for the next meeting of the Central Tennessee CMTA Branch on Monday, August 26th at 6 pm Central Time when we will gather virtually to discuss what to do after receiving a diagnosis of CMT. The session will cover the essential information on available resources, treatment options, and practical strategies […]
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…
Repurposing Drugs for CMT1A: An Encouraging Approach
…nerve cells will alleviate CMT symptoms and slow disease progression. This innovative approach to CMT1A treatment involves repurposing an existing FDA-approved drug, which could expedite the availability of a new…
Summer 2024 CMTA Report: A Season of Renewal and Progress
…intelligence revolutionizes CMT diagnosis, treatment, and management with a dedicated feature by CMTA Chief Research Officer Katherine Forsey, PhD, on page 20 in her column, Under the Microscope. Additionally, community…
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’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…
The Spring 2023 CMTA Report
…of interest: New CMT1X Mouse Model Patients as Partners in Research Tour of Conklin Lab Life-Changing Pharnext Moves Px3003 Production to Us ASO Treatment for CMT1A Circumnavigating the Globe New…
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 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…