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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…
The Fall 2020 CMTA Report
…CMT Join Tribe of the Funky Feet Online CMT1A, CMT1B, CMT4B Projects Approved CMT1A Research Agenda Fuller than Ever 1A Timeline: CMTA Roadmap to Success Consensus Statement on Surgical Treatment…
Find Research by My CMT Type/Subtype
…so essentially the abnormal protein is blocking the function of the working protein and somehow treatment will need to silence that abnormal copy or cut it out. If it can…
The CMT&ME Study: What It’s Like to Live with CMT
…how treatment can improve patient quality of life and slow CMT progression. The study collects data directly from patients using a digital app, CMT&Me, which collects real-world data using “bring…
CMTA Staff
Dr. Suzanne (Sue) Bruhn | she/her Chief Executive Officer Sue has dedicated her career to patient-focused drug development for the treatment of rare diseases. She’s had a long career in…