• CMT1A Ionis Press Release
     New Research Shows Promise for CMT Progressive Neuromuscular Disease Potential treatment offers hope for over 2.8 million affected worldwide Glenolden PA, December 4, 2017—A new paper published December 4th on the Journal of Clinical Investigation (JCI) website ...
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  • CMT1A Research Breakthrough!
    A paper published December 4th on the Journal of Clinical Investigation (JCI) website reveals an exciting potential treatment for patients with Charcot-Marie-Tooth (CMT), the progressive neuromuscular disease that affects more than 2.8 million people ...
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  • Frequently Asked Questions: Ionis/CMTA 1A Research Announcement
    What is CMT1A? Type1ACMT1A is the most common form of Charcot-Marie-Tooth, comprising around 66 percent of all patients with CMT Type 1. The disease is caused by a duplication of the PMP22 gene on Chromosome 17. ...
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  • New Gene Therapy Development Program for CMT2A
    Passage Bio, a genetic medicines company developing AAV-delivered gene therapies, today announced a gene therapy development program for Charcot-Marie-Tooth Neuropathy Type 2A (CMT2A).
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