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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…
CMT Type 2
…the function of the working protein and somehow treatment will need to silence that abnormal copy or cut it out. If it can be silenced, blocked or cut it out…
Keep Hope Alive for Everyone Living with CMT
…treatments are found to prevent/stop progression of CMT and that a cure will soon be within reach. But hope is more than just optimism: it is based on an expectation…
CMTA and Taysha Gene Therapies Announce Collaboration for CMT4A
…Gene Therapies (Nasdaq: TSHA), a patient-centric, pivotal-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS)…
CMTA and ToolGen Announce CMT1A Collaboration
CMTA and ToolGen Announce Collaboration to Develop Potential Treatment for CMT1A Using Gene Therapy (Glenolden, PA, October 7, 2021) The Charcot–Marie–Tooth Association (CMTA), the largest philanthropic funder of CMT research…
CMTA and Addex Announce CMT1A Collaboration
CMTA and Addex Therapeutics Enter Collaboration to Advance GABA B PAM as Potential Treatment for CMT1A Addex Therapeutics is a clinical-stage pharmaceutical company pioneering allosteric modulation-based drug discovery and development….
How to Contact Your Hometown Media
…be identified for early treatment. Charcot-Marie-Tooth is a progressive disorder of the peripheral nerves that causes people to lose the normal use of their feet and legs. Symptoms include: foot…
Pharnext Premier PXT3003 for CMT1A Phase 3 Study
…for CMT1A, and treatment consists of supportive care such as orthoses, physical and occupational therapy or surgery. The purpose of the PREMIER study is to discover if PXT3003 is effective…
The Summer 2021 CMTA Report
…Gray Celebrating Steve (and his 2 decades of fundraising) CMTA Awards Züchner $300,000 for Gene Discovery Work CMTA Awards $100,000 to Explore Potential 1X Treatment CMTA-STAR Awards $1.1 Million in…
All Other Types
…CMT to determine which types may be candidates for treatment with HDAC6 inhibitors and whether HDAC6 inhibitors may be of therapeutic benefit across a variety of CMT types. PROJECT GOAL:…